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Research

Leading-Edge Advancements in Craniofacial Care

The Children's Hospital at Montefiore is part of one of the nation's premier academic medical centers, internationally recognized as a leader of science-driven, patient-centered care, research and education. We have pioneered some of the most innovative and exciting techniques and methodology in the past decade.

All of our craniofacial surgeons were involved with the basic research that makes innovative medical treatments and techniques possible. We have a sophisticated knowledge of how advanced treatments can be best used to benefit each individual child. 

Our team has helped thousands of children who were born with craniofacial differences, and those who have suffered craniofacial trauma. We have developed particular expertise through the treatment of more than 8,000 patients with cleft lip and palate, craniosynostosis and other complex conditions.

Improved Craniofacial Technology and Technique

One of the most thrilling developments in craniofacial care has been the techniques of distraction osteogenesis. First used to lengthen short limbs, this technology can now be applied to the bones of the face, including the lower jaw, mid-face and cranium. These new techniques have vastly improved the outcomes we are able to achieve.

Another area where new techniques have been used is in the realm of bone fixation. Because of concerns regarding growth, we no longer use the metal wires, plates and screws that are used in surgeries performed on the skull. These plates were once typically left in place for a child's entire life, and while problems did not generally arise, surgeons were concerned about potential, as-yet-unknown side effects from this treatment method. Small plates and screws have since been developed that actually dissolve after bone healing has occurred.

We are currently conducting research and clinical trials into the genetic pathways that produce craniofacial deformities, in order to eventually prevent them, and when they cannot be avoided, to help physicians better prepare for their occurrence at the time of a child's birth.

 

Active Clinical Trials

Trial Description
Disease / Condition
Principal Investigator
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Trial DescriptionA 12-Week Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of RO7017773 in Participants Aged 15-45 Years With Autism Spectrum Disorder (ASD) - This study will investigate the efficacy, safety, tolerability, and pharmacokinetics of RO7017773 in participants aged 15-45 years who have been diagnosed with ASD with a score of >/=50 on the Wechsler Abreviated Scale of Intelligence (WASI-II).NCT04299464
Disease/Condition -
Principal InvestigatorEricHollander
Trial DescriptionA 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease - TARGET-IBD is a 5-year, longitudinal, observational study of adult and pediatric patients (age 2 and above) being managed for Inflammatory Bowel Disease (IBD) in usual clinical practice. TARGET-IBD will create a research registry of patients with IBD within academic and community real-world practices in order to assess the safety and effectiveness of current and future therapies.NCT03251118
Disease/Condition -
Principal InvestigatorGititTomer
Trial DescriptionA Longitudinal Observational Study of Patients Undergoing Therapy for IMISC - TARGET-DERM is a longitudinal, observational study of adult and pediatric patients being managed for Atopic Dermatitis and other Immune-Mediated Inflammatory Skin Conditions (IMISC) in usual clinical practice. TARGET-DERM will create a research registry of patients with IMISC within academic and community real-world practices in order to assess the safety and effectiveness of current and future therapies.NCT03661866
Disease/Condition -
Principal InvestigatorHollyKanavy
Trial DescriptionA Longitudinal Observational Study of Patients With Nonalcoholic Steatohepatitis (NASH) and Related Conditions Across the Entire Spectrum of Nonalcoholic Fatty Liver Disease (NAFLD) - TARGET-NASH is a longitudinal observational cohort study of patients being managed for NASH and related conditions across the entire spectrum NAFLD in usual clinical practice. TARGET-NASH is a research registry of patients with NAFL or NASH within academic and community real-world practices maintained in order to assess the safety and effectiveness of current and future therapies.NCT02815891
Disease/Condition -
Principal InvestigatorPreetiViswanathan
Trial DescriptionA Long-term Extension Study of Apremilast (CC-10004) in Pediatric Subjects From 6 Through 17 Years of Age With Moderate to Severe Plaque Psoriasis - This study was created to provide subjects who complete Week 52 (end of Apremilast Extension Phase) of study CC-10004-PPSO-003 the option to continue to receive open-label apremilast therapy. The study will consist of up to 208 weeks of long-term treatment followed by an 8-week observational follow-up phase.NCT04175613
Disease/Condition -
Principal InvestigatorJuliaGittler
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Trial DescriptionA Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) - This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.NCT01351545
Disease/Condition -
Principal InvestigatorMichelleLee
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Trial DescriptionA Multicenter, Prospective, Long-term, Observational Registry of Pediatric Patients With Inflammatory Bowel Disease - The purpose of this study is to evaluate the long-term safety and clinical status of pediatric patients with Inflammatory Bowel Disease (IBD). Particular attention will be directed to recording safety outcomes reported in association with infliximab and other prescribed IBD therapies. In addition, information on disease status and quality of life will be collected.NCT00606346
Disease/Condition -
Principal InvestigatorGititTomer
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Trial DescriptionA Multi-Institution Study of TGFβ Imprinted, Ex Vivo Expanded Universal Donor NK Cell Infusions as Adoptive Immunotherapy in Combination With Gemcitabine and Docetaxel in Patients With Relapsed or Refractory Pediatric Bone and Soft Tissue - The purpose of this study is to determine if the addition of infusions of a type of immune cell called a "natural killer", or NK cell to the sarcoma chemotherapy regimen GEM/DOX (gemcitabine and docetaxel) can improve outcomes in people with childhood sarcomas that have relapsed or not responded to prior therapies. The goals of this study are: To determine the safety and efficacy of the addition of adoptive transfer of universal donor, TGFβ imprinted (TGFβi), expanded NK cells to the pediatric sarcoma salvage chemotherapeutic regimen gemcitabine/docetaxel (GEM/DOX) for treatment of relapsed and refractory pediatric sarcomas To determine the 6-month progression free survival achieved with this treatment in patients within cohorts of relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma and non-rhabdomyosarcoma soft tissue sarcoma. To identify toxicities related to treatment with GEM/DOX + TGFβi expanded NK cells Participants will receive study drugs that include chemotherapy and NK cells in cycles; each cycle is 21 days long and you can receive up to 8 cycles. Gemcitabine (GEM): via IV on Days 1 and 8 Docetaxel (DOX): via IV on Day 8 Prophylactic dexamethasone: Day 7-9 to prevent fluid retention and hypersensitivity reaction Peg-filgrastim (PEG-GCSF) or biosimilar: Day 9 to help your white blood cell recover and allow more chemotherapy to be given TGFβi NK cells: via IV on Day 12NCT05634369
Disease/Condition -
Principal InvestigatorAliceLee
Trial DescriptionA Peer-Led Intervention to Improve Postpartum Retention in HIV Care - The purpose of this study is to test the efficacy of a theory-driven peer intervention for pregnant and postpartum women living HIV. The peer intervention is designed to increase self-efficacy, social support, self-regulatory behaviors, and outcome expectancy in order to improve retention in care and viral suppression postpartum. The intervention will consist of face-to-face prenatal educational sessions, starting in early third trimester, and postpartum sessions scheduled up to three months postpartum.NCT04168008
Disease/Condition -
Principal InvestigatorRodneyWright
Trial DescriptionA Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia - This is a nonrandomized study of ruxolitinib in combination with a standard multi-agent chemotherapy regimen for the treatment of B-cell acute lymphoblastic leukemia. Part 1 of the study will optimize the dose of study drug (ruxolitinib) in combination with the chemotherapy regimen. Part 2 will evaluate the efficacy of combination chemotherapy and ruxolitinib at the recommended dose determined in Part 1.NCT02723994
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease - This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).NCT04293185
Disease/Condition -
Principal InvestigatorDeepaManwani
Trial DescriptionA Study Evaluating Safety and Efficacy of Venetoclax in Combination With Azacitidine Versus Standard of Care After Allogeneic Stem Cell Transplantation (SCT) in Participants With Acute Myeloid Leukemia (AML) - The main objective of this study is to evaluate the efficacy of venetoclax in combination with azacitidine to improve Overall Survival (OS) in Acute Myeloid Leukemia (AML) participants compared to Best Supportive Care (BSC) when given as maintenance therapy following allogeneic stem cell transplantation (SCT). This study will have 2 parts: Part 1 (Dose Confirmation), which may include participants who are greater than or equal to 18 years old; Part 2 (Randomization) which may include participants who are greater than or equal to 12 years old. During Part 1, recommended Phase 3 dose of venetoclax in combination with azacitidine will be determined and during Part 2, the efficacy and safety of venetoclax with azacitidine (Part 2 Arm A) will be compared with BSC (Part 2 Arm B).NCT04161885
Disease/Condition -
Principal InvestigatorIoannisMantzaris
Trial DescriptionA Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery - This phase II trial studies the side effects and how well nirogacestat works in treating patients patients less than 18 years of age with desmoid tumors that has grown after at least one form of treatment by mouth or in the vein that cannot be removed by surgery. Nirogacestat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.NCT04195399
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT - This phase II trial studies the best approach to combine chemotherapy and radiation therapy (RT) based on the patient's response to induction chemotherapy in patients with non-germinomatous germ cell tumors (NGGCT) that have not spread to other parts of the brain or body (localized). This study has 2 goals: 1) optimizing radiation for patients who respond well to induction chemotherapy to diminish spinal cord relapses, 2) utilizing higher dose chemotherapy followed by conventional RT in patients who did not respond to induction chemotherapy. Chemotherapy drugs, such as carboplatin, etoposide, ifosfamide, and thiotepa, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays or high-energy protons to kill tumor cells and shrink tumors. Studies have shown that patients with newly-diagnosed localized NGGCT, whose disease responds well to chemotherapy before receiving radiation therapy, are more likely to be free of the disease for a longer time than are patients for whom the chemotherapy does not efficiently eliminate or reduce the size of the tumor. The purpose of this study is to see how well the tumors respond to induction chemotherapy to decide what treatment to give next. Some patients will be given RT to the spine and a portion of the brain. Others will be given high dose chemotherapy and a stem cell transplant before RT to the whole brain and spine. Giving treatment based on the response to induction chemotherapy may lower the side effects of radiation in some patients and adjust the therapy to a more efficient one for other patients with localized NGGCT.NCT04684368
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT - This phase II trial studies how well combination chemotherapy works in treating patients with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) or favorable histology Wilms tumors (FHWT) that have come back (relapsed). Drugs used in chemotherapy regimens such as UH-3 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan) and ICE/Cyclo/Topo (ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan) work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial may help doctors find out what effects, good and/or bad, regimen UH-3 has on patients with newly diagnosed DAWT and standard risk relapsed FHWT (those treated with only 2 drugs for the initial WT) and regimen ICE/Cyclo/Topo has on patients with high and very high risk relapsed FHWT (those treated with 3 or more drugs for the initial WT).NCT04322318
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) - This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).NCT04624659
Disease/Condition -
Principal InvestigatorDeepaManwani
Trial DescriptionA Study of GSK3511294 (Depemokimab) Compared With Mepolizumab or Benralizumab in Participants With Severe Asthma With an Eosinophilic Phenotype - This study will assess whether switching participants who have benefitted from mepolizumab or benralizumab to GSK3511294 (Depemokimab) is non-inferior to maintaining current treatment on the annualized rate of clinically significant exacerbations in participants with severe asthma with an eosinophilic phenotype. Throughout the study, all participants will continue their non-biologic Baseline standard of care (SoC) asthma treatment.NCT04718389
Disease/Condition -
Principal InvestigatorGoldaHudes
Trial DescriptionA Study of GSK3511294 (Depemokimab) in Participants With Severe Asthma With an Eosinophilic Phenotype - This study will assess the efficacy and safety of GSK3511294 (Depemokimab) as an adjunctive therapy in participants with severe uncontrolled asthma with an eosinophilic phenotype.NCT04718103
Disease/Condition -
Principal InvestigatorGoldaHudes
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Trial DescriptionA Study of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee - The objective of this study is to compare the efficacy and safety of MACI® vs arthroscopic microfracture in the treatment of patients aged 10 to 17 years with symptomatic articular chondral or osteochondral defects of the knee.NCT03588975
Disease/Condition -
Principal InvestigatorEricFornari
Trial DescriptionA Study of Nivolumab Plus Brentuximab Vedotin in Patients Between 5 and 30 Years Old, With Hodgkin's Lymphoma (cHL), Relapsed or Refractory From First Line Treatment - The purpose of this study is to determine whether nivolumab plus brentuximab vedotin (followed by brentuximab vedotin plus bendamustine in patient with suboptimal response) is safe and effective in treating patients with Hodgkin's lymphoma (cHL). Eligible patients are children, adolescents, and young adults relapsed or refractory to first line.NCT02927769
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study of SNDX-5613 in R/R Leukemias Including Those With an MLLr/KMT2A Gene Rearrangement or NPM1 Mutation - Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of SNDX-5613 in participants with acute leukemia. In Phase 2, participants will be enrolled in 3 indication-specific expansion cohorts to determine the efficacy, short- and long-term safety, and tolerability of SNDX-5613.NCT04065399
Disease/Condition -
Principal InvestigatorIoannisMantzaris
Trial DescriptionA Study of the Drug Selinexor With Radiation Therapy in Patients With Newly-Diagnosed Diffuse Intrinsic Pontine (DIPG) Glioma and High-Grade Glioma (HGG) - This phase I/II trial tests the safety, side effects, and best dose of selinexor given in combination with standard radiation therapy in treating children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG) or high-grade glioma (HGG) with a genetic change called H3 K27M mutation. It also tests whether combination of selinexor and standard radiation therapy works to shrink tumors in this patient population. Glioma is a type of cancer that occurs in the brain or spine. Glioma is considered high risk (or high-grade) when it is growing and spreading quickly. The term, risk, refers to the chance of the cancer coming back after treatment. DIPG is a subtype of HGG that grows in the pons (a part of the brainstem that controls functions like breathing, swallowing, speaking, and eye movements). This trial has two parts. The only difference in treatment between the two parts is that some subjects treated in Part 1 may receive a different dose of selinexor than the subjects treated in Part 2. In Part 1 (also called the Dose-Finding Phase), investigators want to determine the dose of selinexor that can be given without causing side effects that are too severe. This dose is called the maximum tolerated dose (MTD). In Part 2 (also called the Efficacy Phase), investigators want to find out how effective the MTD of selinexor is against HGG or DIPG. Selinexor blocks a protein called CRM1, which may help keep cancer cells from growing and may kill them. It is a type of small molecule inhibitor called selective inhibitors of nuclear export (SINE). Radiation therapy uses high energy to kill tumor cells and shrink tumors. The combination of selinexor and radiation therapy may be effective in treating patients with newly-diagnosed DIPG and H3 K27M-Mutant HGG.NCT05099003
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma - This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether selumetinib works better in treating patients with NF1-associated low-grade glioma compared to standard therapy with carboplatin and vincristine.NCT03871257
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss - This phase III trial tests two hypotheses in patients with low-risk and average-risk medulloblastoma. Medulloblastoma is a type of cancer that occurs in the back of the brain. The term, risk, refers to the chance of the cancer coming back after treatment. Subjects with low-risk medulloblastoma typically have a lower chance of the cancer coming back than subjects with average-risk medulloblastoma. Although treatment for newly diagnosed average-risk and low-risk medulloblastoma is generally effective at treating the cancer, there are still concerns about the side effects of such treatment. Side effects or unintended health conditions that arise due to treatment include learning difficulties, hearing loss or other issues in performing daily activities. Standard therapy for newly diagnosed average-risk or low-risk medulloblastoma includes surgery, radiation therapy, and chemotherapy (including cisplatin). Cisplatin may cause hearing loss as a side effect. In the average-risk medulloblastoma patients, this trial tests whether the addition of sodium thiosulfate (STS) to standard of care chemotherapy and radiation therapy reduces hearing loss. Previous studies with STS have shown that it may help reduce or prevent hearing loss caused by cisplatin. In the low-risk medulloblastoma patients, the study tests whether a less intense therapy (reduced radiation) can provide the same benefits as the more intense therapy. The less intense therapy may cause fewer side effects. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of cancer cells. The overall goals of this study are to see if giving STS along with standard treatment (radiation therapy and chemotherapy) will reduce hearing loss in medulloblastoma patients and to compare the overall outcome of patients with medulloblastoma treated with STS to patients treated without STS on a previous study in order to make sure that survival and recurrence of tumor is not worsened.NCT05382338
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study of Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic Arthritis - The purpose of this study is to evaluate the pharmacokinetics (PK), efficacy, safety and immunogenicity of ustekinumab and guselkumab in active juvenile psoriatic arthritis (jPsA).NCT05083182
Disease/Condition -
Principal InvestigatorDawnWahezi
Trial DescriptionA Study to Assess Contraceptive Efficacy and Safety of Etonogestrel (ENG) Implant Beyond 3 Years of Use (MK-8415-060) - The primary purpose of this study is to assess the efficacy and safety of the etonogestrel (ENG) contraceptive implant during participants' fourth and fifth years of use when used as the only method of contraception. The ENG implant is currently approved for a 3-year duration, and this study aims to confirm available evidence suggesting that the ENG implant remains highly effective when used up to 5 years.NCT04626596
Disease/Condition -
Principal InvestigatorJessicaAtrio
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Trial DescriptionA Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib in Participants With Non-Cystic Fibrosis Bronchiectasis - The primary objective of this study is to evaluate the effect of brensocatib at 10 mg and 25 mg compared with placebo on the rate of pulmonary exacerbations (PEs) over the 52-week treatment period.NCT04594369
Disease/Condition -
Principal InvestigatorDivyaReddy
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Trial DescriptionA Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH) - This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of macitentan in children with pulmonary arterial hypertension (PAH).NCT02932410
Disease/Condition -
Principal InvestigatorNicoleSutton
Trial DescriptionA Study to Compare Early Use of Vinorelbine and Maintenance Therapy for Patients With High Risk Rhabdomyosarcoma - This phase III trial compares the safety and effect of adding vinorelbine to vincristine, dactinomycin, and cyclophosphamide (VAC) for the treatment of patients with high risk rhabdomyosarcoma (RMS). High risk refers to cancer that is likely to recur (come back) after treatment or spread to other parts of the body. This study will also examine if adding maintenance therapy after VAC therapy, with or without vinorelbine, will help get rid of the cancer and/or lower the chance that the cancer comes back. Vinorelbine and vincristine are in a class of medications called vinca alkaloids. They work by stopping cancer cells from growing and dividing and may kill them. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy. It works by damaging the cell's deoxyribonucleic acid (DNA) and may kill cancer cells. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's DNA and may kill cancer cells. It may also lower the body's immune response. Vinorelbine, vincristine, dactinomycin and cyclophosphamide are chemotherapy medications that work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial may have the potential to eliminate rhabdomyosarcoma for a long time or for the rest of patient's life.NCT04994132
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations - This phase III trial compares standard chemotherapy to therapy with liposome-encapsulated daunorubicin-cytarabine (CPX-351) and/or gilteritinib for patients with newly diagnosed acute myeloid leukemia with or without FLT3 mutations. Drugs used in chemotherapy, such as daunorubicin, cytarabine, and gemtuzumab ozogamicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. CPX-351 is made up of daunorubicin and cytarabine and is made in a way that makes the drugs stay in the bone marrow longer and could be less likely to cause heart problems than traditional anthracycline drugs, a common class of chemotherapy drug. Some acute myeloid leukemia patients have an abnormality in the structure of a gene called FLT3. Genes are pieces of DNA (molecules that carry instructions for development, functioning, growth and reproduction) inside each cell that tell the cell what to do and when to grow and divide. FLT3 plays an important role in the normal making of blood cells. This gene can have permanent changes that cause it to function abnormally by making cancer cells grow. Gilteritinib may block the abnormal function of the FLT3 gene that makes cancer cells grow. The overall goals of this study are, 1) to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, 2) to study the effects, good and/or bad, of adding gilteritinib to AML therapy for patients with high amounts of FLT3/ITD or other FLT3 mutations and 3) to study changes in heart function during and after treatment for AML. Giving CPX-351 and/or gilteritinib with standard chemotherapy may work better in treating patients with acute myeloid leukemia compared to standard chemotherapy alone.NCT04293562
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionA Study to Evaluate Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis - Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Therapies spread over the skin may not be enough to control the AD in trial participants who require systemic anti-inflammatory treatment. This study compares upadacitinib to dupilumab in adolescent and adult participants with moderate to severe AD who have inadequate response to systemic therapies. Adverse events and change in the disease activity will be assessed. Upadacitinib and dupilumab are approved drugs for the treatment of moderate to severe atopic dermatitis (AD). The study is comprised of a 35-day Screening Period, a 16-week treatment period 1 and a 16-week treatment period 2. During period 1, participants are randomly assigned in 1 of 2 groups, called treatment arms to receive upadacitinib Dose A or dupilumab. In Period 2, participants will receive upadacitinib Dose A or Dose B. Approximately 880 adolescent and adult participants ages 12 to 64 with moderate to severe AD who are candidates for systemic therapy will be enrolled at up to 330 sites worldwide. Participants will receive upadacitinib oral tablets once daily or dupilumab as per its label for 32 weeks and followed for 30 days. There may be higher treatment burden for participants in this trial compared to their standard of care . Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.NCT05601882
Disease/Condition -
Principal InvestigatorHollyKanavy
Trial DescriptionA Study to Evaluate Rocatinlimab (AMG 451) in Adolescent Subjects With Moderate-to-severe Atopic Dermatitis (AD) - The purpose of this study is to evaluate the efficacy and safety of rocatinlimab in monotherapy and combination therapy treatment in adolescent subjects.NCT05704738
Disease/Condition -
Principal InvestigatorHollyKanavy
Trial DescriptionA Study to Evaluate Tabelecleucel in Participants With Epstein-barr Virus-associated Diseases - The purpose of this study is to assess the efficacy and safety of tabelecleucel in participants with Epstein-Barr virus (EBV) associated diseases.NCT04554914
Disease/Condition -
Principal InvestigatorDavidLoeb
Trial DescriptionA Study to Evaluate the Efficacy and Safety of Dupilumab in Participants With Allergic Bronchopulmonary Aspergillosis (ABPA) (LIBERTY ABPA AIRED) - The primary objective of the study is to evaluate the efficacy of dupilumab on lung function in participants with Allergic Bronchopulmonary Aspergillosis (ABPA). The secondary objectives of the study are: To evaluate the effects of dupilumab on exacerbations in participants with ABPA To evaluate the effects of dupilumab on ABPA-related exacerbations To evaluate the effects of dupilumab on hospitalization/emergency department (ED)/urgent care visits in participants with ABPA To evaluate the effects of dupilumab on asthma control in participants with ABPA To evaluate the effects of dupilumab on health-related quality of life (HRQoL) in participants with ABPA To evaluate the effects of dupilumab on serum total immunoglobulin E (IgE) and Aspergillus-specific IgE concentrations To evaluate the effects of dupilumab on Fractional exhaled Nitric Oxide (FeNO) levels To evaluate safety and tolerability of dupilumab in participants with ABPA To evaluate dupilumab concentrations in serum and the incidence of anti-dupilumab antibodies in participants with ABPANCT04442269
Disease/Condition -
Principal InvestigatorGoldaHudes
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Trial DescriptionA Study to Evaluate the Long-Term Safety and Efficacy of Upadacitinib (ABT-494) in Participants With Ulcerative Colitis (UC) - This study is designed to evaluate the long-term safety and efficacy of Upadacitinib in participants with ulcerative colitis (UC) who have not responded at the end of the induction period in Study M14-234 Substudy 1, who have had loss of response during the maintenance period of Study M14-234 Substudy 3, or who have successfully completed Study M14-234 Substudy 3.NCT03006068
Disease/Condition -
Principal InvestigatorThomasUllman
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Trial DescriptionA Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD) - Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA Secondary Objective: To determine the single-dose pharmacokinetics (PK) and pharmacodynamics (PD) profile of ferumoxytol in pediatric subjects.NCT03619850
Disease/Condition -
Principal InvestigatorAnnaZolotnitskaya
Trial DescriptionA Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism - The goal of the study is to evaluate the efficacy and safety of crizanlizumab in SCD patients with priapism.NCT03938454
Disease/Condition -
Principal InvestigatorHennyBillett
Trial DescriptionA Study to See if Memantine Protects the Brain During Radiation Therapy Treatment for a Brain Tumor - This phase III trial compares memantine to usual treatment in treating patients with brain tumors that are newly diagnosed or have come back (recurrent). Memantine may block receptors (parts of nerve cells) in the brain known to contribute to a decline in cognitive function. Giving memantine may make a difference in cognitive function (attention, memory, or other thought processes) in children and adolescents receiving brain radiation therapy to treat a primary brain tumor.NCT04939597
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionAccelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours - The purpose of this study is to determine whether accelerated BEP chemotherapy is more effective than standard BEP chemotherapy in males with intermediate and poor-risk metastatic germ cell tumours.NCT02582697
Disease/Condition -
Principal InvestigatorLisaGennarini
Trial DescriptionActive Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors - This phase III trial studies how well active surveillance help doctors to monitor subjects with low risk germ cell tumors for recurrence after their tumor is removed. When the germ cell tumors has spread outside of the organ in which it developed, it is considered metastatic. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. The trial studies whether carboplatin or cisplatin is the preferred chemotherapy to use in treating metastatic standard risk germ cell tumors.NCT03067181
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionACURATE IDE: Safety and Effectiveness Study of ACURATE Valve for Transcatheter Aortic Valve Replacement - To evaluate safety and effectiveness of the ACURATE Transfemoral Aortic Valve System for transcatheter aortic valve replacement (TAVR) in subjects with severe native aortic stenosis who are indicated for TAVR.NCT03735667
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Principal InvestigatorMohamedAzeemLatib
Trial DescriptionAdoptive Cell Therapy Long-term Follow-up (LTFU) Study - This trial will evaluate long term safety of participants who have received GlaxoSmithKline (GSK) adoptive cell therapy for up to 15 years following last adoptive cell therapy infusion.NCT03391778
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Principal InvestigatorDavidLoeb
Trial DescriptionALLIANCE: Safety and Effectiveness of the SAPIEN X4 Transcatheter Heart Valve - The objective of this study is to establish the safety and effectiveness of the Edwards SAPIEN X4 Transcatheter Heart Valve (THV) in subjects with symptomatic, severe, calcific aortic stenosis (AS).NCT05172960
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Principal InvestigatorMohamedAzeemLatib
Trial DescriptionAlloSure Lung Assessment and Metagenomics Outcomes Study - ALAMO is a prospective, multi-center, perspective, registry of patients receiving LungCare™ (AlloSure®-Lung, AlloMap Lung, AlloID™, iBox, HistoMap, and AlloHeme) for surveillance post-transplant. This study aims to investigate the clinical utility of AlloSure®-Lung for surveillance of the spectrum of rejection and infection events in concert with standard of practice (SOP) post-transplant medical care in a robust lung transplant population. The AlloID™ metagenomics NGS plasma test will be collected and performed for research to validate this molecular diagnostic tool versus SOP microbiologic techniques for detection of allograft infection events.NCT05050955
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Principal InvestigatorAliMansour
Trial DescriptionAn Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC) - The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.NCT04185363
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Principal InvestigatorNadiaOvchinsky
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Trial DescriptionAn Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1 - This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit. Approximately 100 patients in the US will be treated as part of this protocolNCT03259633
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Principal InvestigatorNagmaDalvi
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Trial DescriptionAn Open Label Study of CM-AT for the Treatment of Children With Autism - This is a Phase III, open label extension study evaluating the continued safety and efficacy of CM-AT in pediatric patients with autism with all levels of fecal chymotrypsin.NCT02649959
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Principal InvestigatorEricHollander
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Trial DescriptionAn Open Label Study of FT218 in Subjects With Narcolepsy - An Open Label Study to Evaluate Long-Term Safety and Tolerability of a Once Nightly Formulation of Sodium Oxybate for Extended-Release Oral Suspension (FT218) and the ability to switch from twice-nightly immediate release sodium oxybate to once-nightly FT218 for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Subjects with NarcolepsyNCT04451668
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Principal InvestigatorMichaelThorpy
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Trial DescriptionAn Open-Label Extension Study of GSK3511294 (Depemokimab) in Participants Who Were Previously Enrolled in 206713 (NCT04719832) or 213744 (NCT04718103) - The purpose of this open-label 12-month extension study is to continue to characterize the long-term safety, efficacy and immunogenic profile of GSK3511294 (Depemokimab) in participants with severe asthma with an eosinophilic phenotype following completion of clinical studies 206713 or 213744.NCT05243680
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Principal InvestigatorGoldaHudes
Trial DescriptionAn Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia - An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary AtresiaNCT05426733
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Principal InvestigatorNadiaOvchinsky
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Trial DescriptionApixaban for the Acute Treatment of Venous Thromboembolism in Children - To assess the safety and descriptive efficacy of apixaban in pediatric subjects requiring anticoagulation for the treatment of a VTE.NCT02464969
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Principal InvestigatorJenniferDavila
Trial DescriptionAPOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO) - The APOLLO study is being done in an attempt to improve outcomes after kidney transplantation and to improve the safety of living kidney donation based upon variation in the apolipoprotein L1 gene (APOL1). Genes control what is inherited from a family, such as eye color or blood type. Variation in APOL1 can cause kidney disease. African Americans, Afro-Caribbeans, Hispanic Blacks, and Africans are more likely to have the APOL1 gene variants that cause kidney disease. APOLLO will test DNA from kidney donors and recipients of kidney transplants for APOL1 to determine effects on kidney transplant-related outcomes.NCT03615235
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Principal InvestigatorEnverAkalin
Trial DescriptionAssessing Compliance With Mercaptopurine Treatment in Younger Patients With Acute Lymphoblastic Leukemia in First Remission - This randomized phase III trial studies compliance to a mercaptopurine treatment intervention compared to standard of care in younger patients with acute lymphoblastic leukemia that has had a decrease in or disappearance of signs and symptoms of cancer (remission). Assessing ways to help patients who have acute lymphoblastic leukemia to take their medications as prescribed may help them in taking their medications more consistently and may improve treatment outcomes.NCT01503632
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Principal InvestigatorLisaGennarini
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Trial DescriptionAssessing the Efficacy of Virtual Reality Analgesia (VRA) in Pediatric Patients for Pain Control - To evaluate the efficacy of virtual reality analgesia (VRA) for pediatric patients undergoing Botulinum toxin injections (BTI) for spasticity management. Patients will be assigned to one of three groups; one group of subjects will randomly be assigned to VRA intervention using Google Cardboard Virtual reality head- mounted display powered by a iPod touch, a second group of subjects will receive VRA with Oculus Rift, and a third group of subjects will receive no intervention beyond standard sedation, anesthetic, and/or restraint-this group will serve as the control group.NCT03480724
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Principal InvestigatorYuxiChen
Trial DescriptionAssessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution - This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.NCT05044819
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Principal InvestigatorPujaPatel
Trial DescriptionBiomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma - This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglioneuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.NCT00904241
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Principal InvestigatorLisaGennarini
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Trial DescriptionBlinatumomab in Treating Younger Patients With Relapsed B-cell Acute Lymphoblastic Leukemia - This randomized phase III trial studies how well blinatumomab works compared with standard combination chemotherapy in treating patients with B-cell acute lymphoblastic leukemia that has returned after a period of improvement (relapsed). Immunotherapy with blinatumomab may allow the body's immune system to attack and destroy some types of leukemia cells. It is not yet known whether blinatumomab is more effective than standard combination chemotherapy in treating relapsed B-cell acute lymphoblastic leukemia.NCT02101853
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Principal InvestigatorLisaGennarini
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Trial DescriptionBlood Sample Markers of Reproductive Hormones in Assessing Ovarian Reserve in Younger Patients With Newly Diagnosed Lymphomas - This clinical trial studies blood sample markers of reproductive hormones in assessing ovarian reserve in younger patients with newly diagnosed lymphomas. Studying samples of blood from patients with cancer in the laboratory may help measure the effect of curative therapy for lymphoma on ovarian failure.NCT01793233
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Principal InvestigatorLisaGennarini
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Trial DescriptionBortezomib and Sorafenib Tosylate in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia - This randomized phase III trial studies how well bortezomib and sorafenib tosylate work in treating patients with newly diagnosed acute myeloid leukemia. Bortezomib and sorafenib tosylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving bortezomib and sorafenib tosylate together with combination chemotherapy may be an effective treatment for acute myeloid leukemia.NCT01371981
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Principal InvestigatorLisaGennarini
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Trial DescriptionBrentuximab Vedotin and Combination Chemotherapy in Treating Children and Young Adults With Stage IIB, Stage IIIB, IVA, or IVB Hodgkin Lymphoma - This phase III trial studies brentuximab vedotin and combination chemotherapy to see how well they work compared to combination chemotherapy alone in treating children and young adults with stage IIB with bulk, stage IIIB, IVA, or IVB Hodgkin lymphoma. Combinations of biological substances in brentuximab vedotin may be able to carry cancer-killing substances directly to Hodgkin lymphoma cells. Chemotherapy drugs, such as doxorubicin hydrochloride, bleomycin sulfate, vincristine sulfate, etoposide, prednisone, and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known if combination chemotherapy is more effective with or without brentuximab vedotin in treating children with high-risk Hodgkin lymphoma.NCT02166463
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Principal InvestigatorLisaGennarini
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Trial DescriptionBrentuximab Vedotin or Crizotinib and Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage II-IV Anaplastic Large Cell Lymphoma - This partially randomized phase II trial studies how well brentuximab vedotin or crizotinib and combination chemotherapy works in treating patients with newly diagnosed stage II-IV anaplastic large cell lymphoma. Brentuximab vedotin is a monoclonal antibody, called brentuximab, linked to a toxic agent called vedotin. Brentuximab attaches to CD30 positive cancer cells in targeted way and delivers vedotin to kill them. Crizotinib and methotrexate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether brentuximab vedotin and combination chemotherapy is more effective than crizotinib and combination chemotherapy in treating anaplastic large cell lymphoma.NCT01979536
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Principal InvestigatorLisaGennarini
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Trial DescriptionCancer Care Delivery in Adolescent and Young Adult Patients With Acute Lymphoblastic Leukemia - This study investigates cancer care delivery in adolescent and young adult patients with acute lymphoblastic leukemia. Surveying institutions, evaluating delivery of care at the patient level and seeking input from healthcare providers may help doctors increase rates of adherence to National Comprehensive Cancer Network (NCCN) treatment guidelines. It may also improve care for adolescent and young adult patients with acute lymphoblastic leukemia.NCT03204916
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Principal InvestigatorDavidLoeb
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Trial DescriptionCannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD) - This trial aims to study the efficacy and safety of cannabidivarin (CBDV) in children with ASD.NCT03202303
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Principal InvestigatorEricHollander
Trial DescriptionCardiac Biomarkers in Pediatric Cardiomyopathy (PCM Biomarkers) - Cardiomyopathy is a disease of the heart muscle. It is rare, but it can be serious. Cardiomyopathy in children can result in death, disability, heart transplantation or serious heart rhythm disorders. Natural substances in the blood called cardiac biomarkers can be measured in the laboratory and could be a less invasive way (compared to echocardiograms or MRIs) to detect heart dysfunction in children with cardiomyopathy. Little is known about how useful and valid cardiac biomarkers are in the diagnosis and determination of the symptoms in children with cardiomyopathy. The long-term goal of this project is to study how helpful measuring cardiac biomarkers in children with cardiomyopathy is to their doctors in managing the care of these patients as well as improving their overall health. Measures of these cardiac biomarkers could help doctors in determining how best to care for a child with cardiomyopathy, including when to consider heart transplantation as a treatment option.NCT01873976
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Principal InvestigatorDaphneHsu
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Trial DescriptionCBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS) - This trial aims to study the efficacy and safety of cannabidivarin (CBDV) as a treatment for children with PWS.NCT03848481
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Principal InvestigatorEricHollander
Trial DescriptionCerebrotendinous Xanthomatosis (CTX) Prevalence Study - This is an observational, multicenter study to determine the prevalence of Cerebrotendinous Xanthomatosis (CTX) in patient populations diagnosed with early-onset idiopathic bilateral cataracts. Patients who are potentially eligible for study participation will be identified through a chart review of patients who were seen at each study site prior to that site's initiation, or by entering care at the site while the site is participating in the trial.NCT02638220
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Principal InvestigatorNormanMedow
Trial DescriptionChemotherapy and Radiation Therapy in Treating Young Patients With Newly Diagnosed, Previously Untreated, High-Risk Medulloblastoma/PNET - This randomized phase III trial studies different chemotherapy and radiation therapy regimens to compare how well they work in treating young patients with newly diagnosed, previously untreated, high-risk medulloblastoma. Drugs used in chemotherapy, such as vincristine sulfate, cisplatin, cyclophosphamide, and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Radiation therapy uses high-energy x-rays to kill tumor cells. Carboplatin may make tumor cells more sensitive to radiation therapy. It is not yet known which chemotherapy and radiation therapy regimen is more effective in treating brain tumors.NCT00392327
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Principal InvestigatorLisaGennarini
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Trial DescriptionChemotherapy Followed by Radiation Therapy in Treating Younger Patients With Newly Diagnosed Localized Central Nervous System Germ Cell Tumors - This phase II trial studies how well chemotherapy followed by radiation therapy work in treating younger patients with newly diagnosed central nervous system germ cell tumors that have not spread to other parts of the brain, spinal canal, or body (localized). Drugs used as chemotherapy, such as carboplatin, etoposide, and ifosfamide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high-energy x rays to kill tumor cells. Giving chemotherapy followed by radiation therapy may kill more tumor cells.NCT01602666
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Principal InvestigatorLisaGennarini
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Trial DescriptionChemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma - Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.NCT05304585
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Principal InvestigatorLisaGennarini
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Trial DescriptionChildhood Asthma Mentoring Program for Parents - There are 7.1 million children with asthma. Asthma is the cause of 10.5 million missed days of school, 7.5 million outpatient visits, 640,000 ED visits, and 157,000 hospitalization visits in 2008. Recent work has demonstrated that trained peer mentors (individuals from the community) can be effective in reducing hospitalizations for asthmatic children. This study will evaluate the efficacy of technology-driven parent-to-parent mentoring to reduce asthma-related pediatric hospitalizations and emergency department visits.NCT02747706
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Principal InvestigatorDeepaRastogi
Trial DescriptionChronic Kidney Disease in Children Prospective Cohort Study (CKiD) - The Division of Kidney, Urologic, and Hematologic Diseases (DKUHD) of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), in collaboration with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Heart, Lung and Blood Institute (NHLBI) funded a cooperative agreement including two Clinical Coordinating Centers (at Children's Hospital of Philadelphia and at Children's Mercy Hospital in Kansas City), a central biochemistry laboratory (at the University of Rochester) and a Data Coordinating Center (at Johns Hopkins School of Public Health) to conduct a prospective epidemiological study of children with chronic kidney disease (CKD).NCT00327860
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Principal InvestigatorFrederickKaskel
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Trial DescriptionCIBMTR Research Database - The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies. A secondary purpose of the Research Database is to have a comprehensive source of data to study marrow toxic injuries. Objectives: To learn more about what makes stem cell transplants and cellular therapies work well such as: Determine how well recipients recover from their transplants or cellular therapy; Determine how recovery after a transplant or cellular therapy can be improved; Determine how a donor's or recipient's genetics impact recipient recovery after a transplant or cellular therapy; Determine how access to transplant or cellular therapy for different groups of patients can be improved; Determine how well donors recover from the collection procedures.NCT01166009
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Principal InvestigatorMichelleLee
Trial DescriptionCisplatin and Combination Chemotherapy in Treating Children and Young Adults With Hepatoblastoma or Liver Cancer After Surgery - This partially randomized phase II/III trial studies how well, in combination with surgery, cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or hepatocellular carcinoma. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy may kill more tumor cells than one type of chemotherapy alone.NCT03533582
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Principal InvestigatorLisaGennarini
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Trial DescriptionCMV T Cell Immunity in Pediatric Solid Organ Transplant Recipients - CMV infection and disease remain a significant clinical challenge for pediatric solid organ transplant (SOT) recipients. Current prevention strategies are limited to prophylaxis in which antiviral medication is administered for a period of several months or preemption in which close monitoring of CMV viral load from the peripheral blood is performed and treatment is initiated when CMV is detected. Each of these strategies has risks, costs, and limitations associated with it. Recently, assays for measurement of an individual patient's CMV immunity have been developed and are clinically available. One of these is the Viracor CMV T cell Immunity Panel. This flow cytometry based assay is performed on peripheral blood and measures cytokine release in response to CMV antigen stimulation by flow cytometry. The thresholds for this assay that confer protection against CMV infection in pediatric SOT recipients are not known. Defining CMV-specific cell mediated immune response thresholds that confer protection against CMV reactivation could inform patient specific durations of antiviral prophylaxis or pre-emptive surveillance testing. Therefore, the objective of this study is to quantify CMVresponsive T lymphocyte populations by flow cytometry (Viracor CMV T cell Immunity Panel) in pediatric heart, kidney, and liver transplant recipients within the first year of transplantation and to investigate potential threshold values that correlate with protection against CMV infection (DNAemia).NCT03924219
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Principal InvestigatorBetsyHerold
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Trial DescriptionCollecting and Storing Tissue From Young Patients With Cancer - This laboratory study is collecting and storing tissue, blood, and bone marrow samples from young patients with cancer. Collecting and storing samples of tissue, blood, and bone marrow from patients with cancer to study in the laboratory may help doctors learn more about changes that may occur in DNA and identify biomarkers related to cancer.NCT00898755
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Principal InvestigatorPeterCole
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Trial DescriptionCollection of Samples and Data for the National Marrow Donor Program Repository - This protocol will collect blood samples and medical information from patients who have had a bone marrow transplant using cells from an unrelated donor identified through the National Marrow Donor Program (NMDP). The NMDP has two programs in which patients can participate: the Research Database Program and the Research Sample Repository. Patients who have received a bone marrow transplant at the NIH or other institution from an unrelated donor affiliated with the NMDP may be eligible for this study. Participants in the NMDP Research Database program will have medical information about their disease and their transplant sent to the NMDP before and after the transplant and once a year for the rest of their life. The information will be used to help determine how well transplant recipients recover from their transplant, how recovery after a transplant can be improved, how access to transplant for different groups of patients can be improved and how well donors recover from collection procedures. Participants in the NMDP Research Sample Repository program will have a small blood sample drawn from a vein in the arm just before they start taking medicines for the conditioning regimen to prepare them for the transplant. The blood samples will be used to look at ways to improve how patients are matched with their donors, to determine and evaluate the factors that affect transplant outcome, and to help develop methods to improve tissue matching between donors and recipients.NCT00495300
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Principal InvestigatorIraBraunschweig
Trial DescriptionCombination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor - This phase III trial studies how well combination chemotherapy and surgery work in treating young patients with Wilms tumor. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving it after surgery may kill any tumor cells that remain after surgery.NCT00945009
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Principal InvestigatorLisaGennarini
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Trial DescriptionCombination Chemotherapy Followed By Peripheral Stem Cell Transplant in Treating Young Patients With Newly Diagnosed Supratentorial Primitive Neuroectodermal Tumors or High-Risk Medulloblastoma - This randomized phase III trial is studying two different combination chemotherapy regimens to compare how well they work in treating young patients with newly diagnosed supratentorial primitive neuroectodermal tumors or high-risk medulloblastoma when given before additional intense chemotherapy followed by peripheral blood stem cell rescue. It is not yet known which combination chemotherapy regimen is more effective when given before a peripheral stem cell transplant in treating supratentorial primitive neuroectodermal tumors or medulloblastoma.NCT00336024
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Principal InvestigatorJonathanGill
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Trial DescriptionCombination Chemotherapy in Treating Patients With Non-Metastatic Extracranial Ewing Sarcoma - This trial examined the outcome benefit to patients of adding a new chemotherapy drug combination to the established treatment approach for patients with extracranial Ewing sarcoma, that had not spread from the primary site to other places in the body. The trial randomly assigned patients at the time of study entry to receive established standard treatment with the following 5-drugs: vincristine sulfate, doxorubicin hydrochloride, cyclophosphamide, ifosfamide and etoposide. The outcome for patients receiving the standard 5-drug combination was compared to the outcome for patients who received the same 5-drugs with an additional drug, topotecan hydrochloride delivered in a novel combination with vincristine sulfate and cyclophosphamide.NCT01231906
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Principal InvestigatorLisaGennarini
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Trial DescriptionCombination Chemotherapy in Treating Young Patients With Newly Diagnosed High-Risk B Acute Lymphoblastic Leukemia and Ph-Like TKI Sensitive Mutations - This randomized phase III trial studies how well combination chemotherapy works in treating young patients with newly diagnosed B acute lymphoblastic leukemia that is likely to come back or spread, and in patients with Philadelphia chromosome (Ph)-like tyrosine kinase inhibitor (TKI) sensitive mutations. Chemotherapy drugs, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy) and giving the drugs in different doses and in different combinations may kill more cancer cells.NCT02883049
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Principal InvestigatorLisaGennarini
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Trial DescriptionCombination Chemotherapy With or Without Bortezomib in Treating Younger Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or Stage II-IV T-Cell Lymphoblastic Lymphoma - This randomized phase III trial compares how well combination chemotherapy works when given with or without bortezomib in treating patients with newly diagnosed T-cell acute lymphoblastic leukemia or stage II-IV T-cell lymphoblastic lymphoma. Bortezomib may help reduce the number of leukemia or lymphoma cells by blocking some of the enzymes needed for cell growth. It may also help chemotherapy work better by making cancer cells more sensitive to the drugs. It is not yet known if giving standard chemotherapy with or without bortezomib is more effective in treating newly diagnosed T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma.NCT02112916
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Principal InvestigatorLisaGennarini
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Trial DescriptionCombination Chemotherapy With or Without Ganitumab in Treating Patients With Newly Diagnosed Metastatic Ewing Sarcoma - This randomized phase III trial studies how well combination chemotherapy with or without ganitumab works in treating patients with newly diagnosed Ewing sarcoma that has spread to other parts of the body. Treatment with drugs that block the IGF-1R pathway, such as ganitumab, may interfere with the ability of tumor cells to grow and spread. Drugs used in chemotherapy, such as vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether adding ganitumab to combination chemotherapy is more effective in treating patients with newly diagnosed metastatic Ewing sarcoma.NCT02306161
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Principal InvestigatorLisaGennarini
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Trial DescriptionCombination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma - This randomized phase III trial studies how well combination chemotherapy (vincristine sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan hydrochloride or vinorelbine) works compared to combination chemotherapy plus temsirolimus in treating patients with rhabdomyosarcoma (cancer that forms in the soft tissues, such as muscle), and has an intermediate chance of coming back after treatment (intermediate risk). Drugs used work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Combination chemotherapy and temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether chemotherapy plus temsirolimus is more effective than chemotherapy alone in treating patients with intermediate-risk rhabdomyosarcoma.NCT02567435
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Principal InvestigatorLisaGennarini
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Trial DescriptionComparison of Radiation Therapy Regimens in Combination With Chemotherapy in Treating Young Patients With Newly Diagnosed Standard-Risk Medulloblastoma - This randomized phase III trial is studying how well standard-dose radiation therapy works compared to reduced-dose radiation therapy in children 3-7 years of age AND how well standard volume boost radiation therapy works compared to smaller volume boost radiation therapy when given together with chemotherapy in treating young patients who have undergone surgery for newly diagnosed standard-risk medulloblastoma. Radiation therapy uses high-energy x-rays to damage tumor cells. Drugs used in chemotherapy, such as vincristine, cisplatin, lomustine, and cyclophosphamide, work in different ways to stop tumor cells from dividing so they stop growing or die. Giving radiation therapy with chemotherapy after surgery may kill any remaining tumor cells. It is not yet known whether standard-dose radiation therapy is more effective than reduced-dose radiation therapy when given together with chemotherapy after surgery in treating young patients with medulloblastoma.NCT00085735
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Principal InvestigatorPeterCole
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Trial DescriptionCOVID-19 Vaccine Response in Sickle Cell Disease - The purpose of this study is to assess the antibody response to COVID-19 vaccination in a cohort of patients with sickle cell disease (SCD) and to assess vaccine and SCD related complications around the time of vaccination.NCT05139992
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Principal InvestigatorDeepaManwani
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Trial DescriptionCTL019 Out of Specification MAP for ALL or DLBCL Patients - Managed Access Program (MAP) to provide access to CTL019, for acute lymphoblastic leukemia (ALL) or diffuse large b-cell lymphoma (DLBCL) patients with out of specification leukapheresis product and/or manufactured tisagenlecleucel out of specification for commercial release.NCT03601442
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Principal InvestigatorEllenFraint
Trial DescriptionDabrafenib Combined With Trametinib After Radiation Therapy in Treating Patients With Newly-Diagnosed High-Grade Glioma - This phase II trial studies how well the combination of dabrafenib and trametinib works after radiation therapy in children and young adults with high grade glioma who have a genetic change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill tumor cells and reduce the size of tumors. Dabrafenib and trametinib may stop the growth of tumor cells by blocking BRAF and MEK, respectively, which are enzymes that tumor cells need for their growth. Giving dabrafenib with trametinib after radiation therapy may work better than treatments used in the past in patients with newly-diagnosed BRAF V600-mutant high-grade glioma.NCT03919071
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Principal InvestigatorLisaGennarini
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Trial DescriptionDenosumab in Treating Patients With Recurrent or Refractory Osteosarcoma - This phase II trial studies how well denosumab works in treating patients with osteosarcoma that has come back (recurrent) or does not respond to treatment (refractory). Immunotherapy with monoclonal antibodies, such as denosumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.NCT02470091
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Principal InvestigatorLisaGennarini
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Trial DescriptionDevelopmental Impact of NICU Exposures (DINE) - The DINE study will test the hypothesis that potentially avoidable NICU-based exposures contribute to the neuro-cognitive and somatic impairments prevalent among NICU graduates. This hypothesis is drawn from the documented impact of phthalate exposure on early development in term-born children, and the acknowledged presence of these toxic chemicals in the NICU. Third trimester in utero exposure to phthalates have been linked to poorer childhood performance in cognition, motor function, attention, hyperactivity and social behavior. Phthalate exposure is also associated with altered onset of puberty and asthma. The multi-site cohort and approach will clarify the role of NICU-based phthalate exposure on high-prevalence clinical outcomes.NCT03061890
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Principal InvestigatorJudyAschner
Trial DescriptionDinutuximab in Combination With Sargramostim in Treating Patients With Recurrent Osteosarcoma - This phase II trial studies how well dinutuximab works when given with sargramostim in treating patients with osteosarcoma that has come back after treatment (recurrent). Monoclonal antibodies, such as dinutuximab, may find tumor cells and help kill them. Sargramostim may help the body increase the amount of white blood cells it produces, which help the body fight off infections. Giving dinutuximab with sargramostim may work better and kill more cancer cells.NCT02484443
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Principal InvestigatorLisaGennarini
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Trial DescriptionEarly Childhood Obesity Programming by Intrauterine Growth Restriction - The molecular mechanisms underlying developmental programming of childhood obesity remain poorly understood. Here, the investigators address major questions about early childhood obesity programming by studying CD3+ T-cells from intrauterine growth restricted (IUGR) newborns who have an increased risk for obesity and other metabolic disorders in adult life.NCT03402139
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Principal InvestigatorMamtaFuloria
Trial DescriptionEfficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) - Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in children with biliary atresia who have undergone a Kasai hepatoportoenterostomy.NCT04336722
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Principal InvestigatorDebraPan
Trial DescriptionEfficacy and Safety Study of Rimegepant for the Preventative Treatment of Migraine in Pediatric Subjects - The purpose of this study is to compare the efficacy and safety of rimegepant to placebo as a preventative treatment for migraine in children and adolescents ≥ 6 toNCT05156398
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Principal InvestigatorJelenaPavlovic
Trial DescriptionEfficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036) - This study aims to compare the efficacy of vericiguat versus placebo on change in n-terminal pro-brain natriuretic peptide (NTproBNP) from baseline to Week 16. The primary hypothesis is Vericiguat is superior to placebo in reducing NT-proBNP at Week 16.NCT05714085
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Principal InvestigatorDaphneHsu
Trial DescriptionEliminating Monitor Overuse Trial (EMO Trial) - The purpose of this study is to identify the optimal deimplementation strategies for an overused practice: continuous pulse oximetry monitoring of children hospitalized with bronchiolitis who are not receiving supplemental oxygen.NCT05132322
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Principal InvestigatorAlyssaSilver
Trial DescriptionEndocrine Response in Women With Invasive Lobular Breast Cancer - RATIONALE: Currently, adjuvant endocrine therapy often follows a "one-size-fits- all" approach, with most premenopausal women receiving tamoxifen, and most postmenopausal receiving aromatase inhibitor therapy. In current clinical practice, patients with invasive lobular carcinoma are treated no differently than patients with invasive ductal carcinoma based on the void of information specific to patients with this tumor type. Identification of a biological signal of tamoxifen and/or AI-resistance and/or fulvestrant-sensitivity in ILC patients would have dramatic implications for the future management of this breast cancer subtype. PURPOSE: To study whether fulvestrant is more effective than anastrozole or tamoxifen in reducing Ki67 in ILC and whether that Ki67 reduction will correlate with alterations in expression of ER and ER-regulated genes. Differential Ki67 effect in this study will serve as a surrogate for outcome of ILC patients on endocrine therapy. Primary Objective: To determine the change from baseline to post-treatment Ki67 values in ER-positive, HER2-negative ILC tissue derived from postmenopausal women awaiting definitive surgery or further neoadjuvant treatment who are randomized to 21-24 days of neoadjuvant endocrine treatments with fulvestrant (two 250 mg IM injections given on day 1), anastrozole (1mg given orally daily), or tamoxifen (20mg given orally daily).NCT02206984
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Principal InvestigatorSheldonFeldman
Trial DescriptionEnsartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial) - This phase II Pediatric MATCH trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back (recurrent) or does not respond to treatment (refractory) and may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced). Ensartinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.NCT03213652
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Principal InvestigatorLisaGennarini
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Trial DescriptionErdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial) - This phase II Pediatric MATCH trial studies how well erdafitinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have spread to other places in the body and have come back or do not respond to treatment with FGFR mutations. Erdafitinib may stop the growth of cancer cells with FGFR mutations by blocking some of the enzymes needed for cell growth.NCT03210714
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Principal InvestigatorLisaGennarini
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Trial DescriptionEvaluating the Impact of Patient Photographs for Preventing Wrong-Patient Errors - This is a multi-site, cluster-randomized controlled trial to test the effectiveness of patient photographs displayed in electronic health record (EHR) systems to prevent wrong-patient order errors. The study will be conducted at three academic medical centers that utilize two different EHR systems. Because EHR systems have different functionality for displaying patient photographs, two different study designs will be employed. In Allscripts EHR, a 2-arm randomized trial will be conducted in which providers are randomized to view order verification alerts with versus without patient photographs when placing electronic orders. In Epic EHR, a 2x2 factorial trial will be conducted in which providers are randomized to one of four conditions: 1) no photograph; 2) photograph displayed in the banner only; 3) photograph displayed in a verification alert only; or 4) photograph displayed in the banner and verification alert. The main hypothesis of this study is that displaying patient photographs in the EHR will significantly reduce the frequency of wrong-patient order errors, providing health systems with the evidence needed to adopt this safety practice. We will use the Wrong-Patient Retract-and-Reorder (RAR) measure, a valid, reliable, and automated method for identifying wrong-patient orders, as the primary outcome measure. The RAR measure identifies orders placed for a patient that are retracted within 10 minutes, and then reordered by the same provider for a different patient within the next 10 minutes. These are near-miss errors, self-caught by the provider before they reach the patient and cause harm. In one study, the RAR measure identified more than 5,000 wrong-patient orders in 1 year, with a rate of 58 wrong-patient errors per 100,000 orders. Real-time telephone interviews with clinicians determined that the RAR measure correctly identified near-miss errors in 76.2% of cases. Thus, the RAR measure provides sufficient valid and reliable outcome data for this study.NCT03626766
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Principal InvestigatorWilliamSouthern
Trial DescriptionEvaluation of Feeding Intolerance in Premature Infants Using Near Infrared Spectroscopy - Nowadays feeding intolerance (FI) is a common condition among preterm infants. It has been estimated that 16%-29% of premature infants admitted to neonatal intensive care units (NICUs) develop feeding intolerance at some point during their length of stay. The most frequent signs of FI are the presence of abdominal distension, abundant and/or bilious gastric residuals and vomiting suggesting an inability of the infant to further tolerate enteral nutrition, it increases with decreasing in gestational age (GA) and birth weight (BW). FI represents one of the most uncontrollable variables in the early nutritional management of these infants, and may lead to suboptimal nutrition, delayed attainment of full enteral feeding and prolonged parenteral nutrition supply. NIRS has been used in preterm infants to evaluate changes in cerebral perfusion and oxygenation. It provides real time insight into the oxygen delivery, presented as regional oxygen saturation rSO2 with lower values than SpO2 distal pulse-oximetry where is mostly measured as arterialized capillary bed (around 55% vs 98% Oxygen saturation in regional NIRS vs conventional pulse-oximetry). Light easily penetrates the thin tissues of the neonate through bone and soft tissue, particularly the thin capillary bed of the tissues; NIRS provides non-invasive, continuous information on tissue perfusion and oxygen dynamics. This technique uses principles of optical spectrophotometry that make use of the fact that biological material, including the skull, is relatively transparent in the NIR range. Dave et al. evaluated the abdominal tissue oxygenation with NIRS, and showed that preterm infants change their cerebral - splanchnic oxygenation ratios during feedings, mainly because an increasing in the splanchnic oxygenation. Gay et al. performed abdominal NIRS in premature piglets showing association of perfusion/oxygen changes with NEC spectrum. The investigators would like to evaluate the association between feeding intolerance and unchanged splanchnic regional saturation and variation in the cerebral splanchnic ratio. Innovation: FI diagnosis follows a subjective approach, where the clinician is worried in further risk of develop Necrotizing enterocolitis (NEC). This non-studied relationship (FI and NEC) lower the threshold for the diagnosis of FI. Furthermore, infants with FI diagnosis commonly are subject of stop or slow the progression of feedings, increasing the risk of intestinal villi atrophy, and increase the length of parenteral nutrition support, and also the length of stay in the NICU settings. If NIRS technology help the clinicians to detect true abnormalities objectively as a new monitor assessing adequate feeds progress decreasing failure to feed, and therefore diminishing the need for parenteral feeds and further complication associated with it.NCT02534090
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Principal InvestigatorSuhasNafday
Trial DescriptionEvaluation of Maralixibat in Biliary Atresia Response Post-Kasai - A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).NCT04524390
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Principal InvestigatorLidijaPalezac
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Trial DescriptionEvaluation of the Asthma Management Program to Promote Activity for Students in Schools (Asthma-PASS) - Physical activity (PA) is an important component of asthma management in children. Studies show that PA is associated with decreased severity of asthma symptoms, as well as improved disease control and quality of life. However, urban minority children with asthma face barriers to PA on multiple levels.The goal of this research project is to evaluate whether a multifaceted school-based intervention that addresses key barriers to physical activity reduces asthma morbidity among urban schoolchildren with asthma.NCT04576442
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Principal InvestigatorMarinaReznik
Trial DescriptionEvaluation of Upadacitinib in Adolescent and Adult Patients With Moderate to Severe Atopic Dermatitis (Eczema) - The objective of this study is to assess the efficacy and safety of upadacitinib for the treatment of adolescent and adult participants with moderate to severe atopic dermatitis (AD) who are candidates for systemic therapy.NCT03569293
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Principal InvestigatorHollyKanavy
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Trial DescriptionEvolutionary Therapy for Rhabdomyosarcoma - This clinical trial will evaluate 4 different strategies of chemotherapy schedules in newly diagnosed participants with metastatic Fusion Positive (alveolar) Rhabdomyosarcoma. The participant and their physician will choose from: Arm A) a first strike therapy, Arm B) a first strike-second strike (maintenance) therapy, Arm C) an adaptively timed therapy, and Arm D) conventional chemotherapy.NCT04388839
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Principal InvestigatorAliceLee
Trial DescriptionExamining Genetic Factors That Affect the Severity of 22q11.2 Deletion Syndrome - 22q11.2 deletion syndrome is a genetic disorder that can cause heart defects, facial abnormalities, and developmental and learning disabilities. The severity of the disorder can vary widely among people. This study will analyze DNA from people with 22q11.2 deletion syndrome to identify genetic variations that may affect the severity of the disorder.NCT00556530
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Principal InvestigatorBerniceMorrow
Trial DescriptionExtension Study in Primary Distal Renal Tubular Acidosis - Open-label study involving longitudinal assessment of the continued safety, tolerability, and efficacy of ADV7103 in maintaining targeted serum bicarbonate levels, preventing metabolic acidosis, and preventing hypokalemia in the following groups of subjects with primary dRTA: subjects who participated in Study B23CS and were adherent to the protocol; subjects ≥ 6 months of age who are living in Europe and did not participate in Study B23CS; and infants younger than 6 months of ageNCT03831152
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Principal InvestigatorKimberlyReidy
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Trial DescriptionFerric Citrate and Chronic Kidney Disease in Children - We will conduct a 12-month, double-blind, randomized, placebo-controlled trial to assess the effects of therapy with ferric citrate (FC) on changes in intact FGF23 levels (iFGF23, primary endpoint) in 160 pediatric patients (80 in each of the two arms) aged 6-17 years of either sex with chronic kidney disease (CKD) stages 3-4 and age-appropriate normal serum phosphate levels. Participants will be randomized to one of the two groups: 1) FC or 2) FC placebo. Participants will be recruited from 12 core clinical sites.NCT04741646
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Principal InvestigatorFrederickKaskel
Trial DescriptionFirst-in-human Study of SAR443579 Infusion in Male and Female Participants of at Least 12 Years of Age With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL) or High Risk-myelodysplasia (HR-MDS) - This is an open-label, multicenter, Phase 1/Phase 2, dose escalation and dose expansion study to evaluate the safety, pharmacokinetics, pharmacodynamics and anti-leukemic activity of SAR443579 in various hematological malignancies.NCT05086315
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Principal InvestigatorIoannisMantzaris
Trial DescriptionIBP-9414 for the Prevention of Necrotizing Enterocolitis - The Connection Study - IBP-9414 will be evaluated in preterm infants with a birth weight of 500-1500g, compared to placebo with regards to efficacy and safety in the prevention of necrotizing enterocolitis.NCT03978000
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Principal InvestigatorThomasHavranek
Trial DescriptionIbuprofen Plus Acetaminophen for Enhanced Pain Reduction - Combination of analgesics with different modes of action have the potential to offer enhanced pain relief with reduced dosage. Ibuprofen and acetaminophen have different modes of action. Combination of ibuprofen and acetaminophen has shown enhanced analgesia in adult studies, with pediatric data limited to post-operative pain and dental pain. Our study objective is to evaluate the analgesic efficacy of oral acetaminophen plus ibuprofen versus ibuprofen plus placebo for the management of acute pain in a pediatric emergency department.NCT04630834
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Principal InvestigatorSandraCunningham
Trial DescriptionImatinib Mesylate and Combination Chemotherapy in Treating Patients With Newly Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia - This randomized phase III trial studies how well imatinib mesylate works in combination with two different chemotherapy regimens in treating patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia (ALL). Imatinib mesylate has been shown to improve outcomes in children and adolescents with Philadelphia chromosome positive (Ph+) ALL when given with strong chemotherapy, but the combination has many side effects. This trial is testing whether a different chemotherapy regimen may work as well as the stronger one but have fewer side effects when given with imatinib. The trial is also testing how well the combination of chemotherapy and imatinib works in another group of patients with a type of ALL that is similar to Ph+ ALL. This type of ALL is called "ABL-class fusion positive ALL", and because it is similar to Ph+ ALL, is thought it will respond well to the combination of agents used to treat Ph+ ALL.NCT03007147
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Principal InvestigatorLisaGennarini
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Trial DescriptionImmunotherapy (Nivolumab or Brentuximab Vedotin) Plus Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage III-IV Classic Hodgkin Lymphoma - This phase III trial compares immunotherapy drugs (nivolumab or brentuximab vedotin) when given with combination chemotherapy in treating patients with newly diagnosed stage III or IV classic Hodgkin lymphoma. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Brentuximab vedotin is a monoclonal antibody, brentuximab, linked to a toxic agent called vedotin. Brentuximab attaches to cancer cells in a targeted way and delivers vedotin to kill them. Chemotherapy drugs, such as doxorubicin, vinblastine, and dacarbazine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. The addition of nivolumab or brentuximab vedotin to combination chemotherapy may shrink the cancer or extend the time without disease symptoms coming back.NCT03907488
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Principal InvestigatorLisaGennarini
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Trial DescriptionImmunotherapy Following Reduced Intensity Conditioning and Allogeneic Stem Cell Transplant for Poor Risk CD30+ Hodgkin Lymphoma Patients - Patients with relapsed or refractory Hodgkin Lymphoma who are CD30+ will receive a standard of care reduced intensity regimen and an allogeneic stem cell transplant (from another person, related or unrelated). Following recovery, patients will receive a medication called Brentuximab Vendotin which is targeted against CD30+ cells. The study hypothesis is that this treatment will be safe and well tolerated in children and young adults.NCT02098512
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Principal InvestigatorKrisMahadeo
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Trial DescriptionImplementing Models for Mechanical Circulatory Support Presurgical Assessment in Congenital Heart Disease Treatment - The purpose of this research study is to look at the advantages of using a 3D printed heart model for surgical planning in children who have been diagnosed with Congenital Heart Disease (CHD) and clinical heart failure and will undergo a ventricular assist device (VAD) placement. The investigators want to study the correlation of having a 3D printed model with improvement in patient outcomes and compare those with patients who have had a VAD placement without a 3D model.NCT03891160
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Principal InvestigatorOmarSaeed
Trial DescriptionImproving Patient-Centered Communication in Breast Cancer Through Patient and Provider Interventions - This trial studies the effectiveness of two interventions on patient reported outcomes of patient centered communication and decision making about breast cancer treatment. The first intervention consists of enhancements to an existing patient-facing breast cancer treatment decision tool called iCanDecide that supports the management of worry, distress, and anxiety as compared to an existing tool. The second intervention consists of a clinician dashboard that populates information after patients view either website regarding any ongoing issues or concerns.NCT04549571
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Principal InvestigatorSheldonFeldman
Trial DescriptionInotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy - This phase III trial studies whether inotuzumab ozogamicin added to post-induction chemotherapy for patients with High-Risk B-cell Acute Lymphoblastic Leukemia (B-ALL) improves outcomes. This trial also studies the outcomes of patients with mixed phenotype acute leukemia (MPAL), and B-lymphoblastic lymphoma (B-LLy) when treated with ALL therapy without inotuzumab ozogamicin. Inotuzumab ozogamicin is a monoclonal antibody, called inotuzumab, linked to a type of chemotherapy called calicheamicin. Inotuzumab attaches to cancer cells in a targeted way and delivers calicheamicin to kill them. Other drugs used in the chemotherapy regimen, such as cyclophosphamide, cytarabine, dexamethasone, doxorubicin, daunorubicin, methotrexate, leucovorin, mercaptopurine, prednisone, thioguanine, vincristine, and pegaspargase or calaspargase pegol work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial will also study the outcomes of patients with mixed phenotype acute leukemia (MPAL) and disseminated B lymphoblastic lymphoma (B-LLy) when treated with high-risk ALL chemotherapy. The overall goal of this study is to understand if adding inotuzumab ozogamicin to standard of care chemotherapy maintains or improves outcomes in High Risk B-cell Acute Lymphoblastic Leukemia (HR B-ALL). The first part of the study includes the first two phases of therapy: Induction and Consolidation. This part will collect information on the leukemia, as well as the effects of the initial treatment, in order to classify patients into post-consolidation treatment groups. On the second part of this study, patients will receive the remainder of the chemotherapy cycles (interim maintenance I, delayed intensification, interim maintenance II, maintenance), with some patients randomized to receive inotuzumab. Other aims of this study include investigating whether treating both males and females with the same duration of chemotherapy maintains outcomes for males who have previously been treated for an additional year compared to girls, as well as to evaluate the best ways to help patients adhere to oral chemotherapy regimens. Finally, this study will be the first to track the outcomes of subjects with disseminated B-cell Lymphoblastic Leukemia (B-LLy) or Mixed Phenotype Acute Leukemia (MPAL) when treated with B-ALL chemotherapy.NCT03959085
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Principal InvestigatorLisaGennarini
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Trial DescriptionIntegrated Community Based Health Systems Strengthening in Northern Togo - The general objective of this study is to optimize implementation and assess effectiveness of the integrated facility and community-based health systems strengthening (ICBHSS) model in four Northern Togo districts, using the RE-AIM implementation science framework. Specific study aims include: (1) Analyze longitudinal changes regarding maternal and child health outcomes, health service utilization rates, and public sector facility readiness in the ICBHSS model intervention sites catchment areas; (2) Identify barriers to and facilitators of access and quality services related to ICBHSS model; and (3) Assess changes in health care services coverage, effectiveness, and adoption of ICBHSS model. These findings are expected to contribute to continuous quality improvement initiatives, optimize implementation factors, provide generalizable knowledge regarding health service delivery, and accelerate health systems improvements in Togo and more broadly.NCT03694366
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Principal InvestigatorKevinFiori
Trial DescriptionIntegrated Community Based Health Systems Strengthening Preliminary Study in Kozah Togo - This preliminary study aims to inform the design and delivery of the integrated facility and community-based health systems strengthening (ICBHSS) model in four Kozah District health facilities over a period of 48 months. Specific aims include: (1) Assess maternal and child health outcomes and health service utilization rates in the 4 ICBHSS model intervention sites catchment areas; (2) Identify barriers to and facilitators of access and quality services related to ICBHSS model; and (3) Assess changes in health care services coverage, effectiveness, and adoption of ICBHSS model.NCT03773913
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Principal InvestigatorKevinFiori
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Trial DescriptionIntergroup Trial for Children or Adolescents With Primary Mediastinal Large B-Cell Lymphoma: DA-EPOCH-Rituximab Evaluation - Phase II trial to determine the efficacy of Dose Adjusted-EPOCH-Rituximab regimen in children and adolescent with primary mediastinal large B cell lymphoma in terms of event free survival.NCT01516567
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Principal InvestigatorJonathanGill
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Trial DescriptionIntranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome - This study is a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2) repetitive behaviors (3) weight and body composition (4) quality of life (5) salivary OXT and hormone levels (including ghrelin, pancreatic polypeptide, peptide YY, GLP-1, insulin, glucagon, testosterone, and estrogen). If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS. Funding Source- FDA OOPDNCT03197662
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Principal InvestigatorEricHollander
Trial DescriptionIntravenous Gammaglobulin for Sickle Cell Pain Crises - The purpose of this study is to determine whether intravenous immune globulin is safe and effective in the acute treatment of pain crises in sickle cell disease. Funding Source: Food and Drug Administration (FDA), Office of Orphan Products Development (OOPD)NCT01757418
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Principal InvestigatorDeepaManwani
Trial DescriptionInvestigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults - The primary objective is to evaluate the efficacy of carisbamate (YKP509) as adjunctive treatment in reducing the number of drop seizures (tonic, atonic, and tonic-clonic) compared with placebo in pediatric and adult subjects (age 4-55 years) diagnosed with Lennox Gastaut Syndrome (LGS).NCT05219617
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Principal InvestigatorAlexisBoro
Trial DescriptionInvestigating Dupilumab's Effect in Asthma by Genotype - The Goal of this study is to investigate if individuals ages 12 years and older, carrying the IL-4RαR576 gene variant, will have a greater response to therapy acting directly on the anti-IL-4R. This will be conducted by examining the effect of a 48 week therapy with dupilumab on the rate of asthma exacerbations.NCT03694158
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Principal InvestigatorSunitJariwala
Trial DescriptionIobenguane I-131 or Crizotinib and Standard Therapy in Treating Younger Patients With Newly-Diagnosed High-Risk Neuroblastoma or Ganglioneuroblastoma - This phase III trial studies iobenguane I-131 or lorlatinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Lorlatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving iobenguane I-131 or lorlatinib and standard therapy may work better compared to lorlatinib and standard therapy alone in treating younger patients with neuroblastoma or ganglioneuroblastoma.NCT03126916
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Principal InvestigatorLisaGennarini
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Trial DescriptionIpilimumab or High-Dose Interferon Alfa-2b in Treating Patients With High-Risk Stage III-IV Melanoma That Has Been Removed by Surgery - This randomized phase III trial studies ipilimumab to see how well it works compared to high-dose interferon alfa-2b in treating patients with high-risk stage III-IV melanoma that has been removed by surgery. Immunotherapy with monoclonal antibodies, such as ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Interferon alfa-2b may interfere with the growth of tumor cells and slow the growth of melanoma and other cancers. It is not yet known whether ipilimumab is more effective than interferon alfa-2b in treating patients with melanoma.NCT01274338
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Principal InvestigatorJosephSparano
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Trial DescriptionIrinotecan Hydrochloride, Temozolomide, and Dinutuximab With or Without Eflornithine in Treating Patients With Relapsed or Refractory Neuroblastoma - This phase II trial studies how well irinotecan hydrochloride, temozolomide, and dinutuximab work with or without eflornithine in treating patients with neuroblastoma that has come back (relapsed) or that isn't responding to treatment (refractory). Drugs used in chemotherapy, such as irinotecan hydrochloride and temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Immunotherapy with monoclonal antibodies, such as dinutuximab, may induce changes in the body's immune system and may interfere with the ability of tumor cells to grow and spread. Eflornithine blocks the production of chemicals called polyamines that are important in the growth of cancer cells. Giving eflornithine with irinotecan hydrochloride, temozolomide, and dinutuximab, may work better in treating patients with relapsed or refractory neuroblastoma.NCT03794349
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Principal InvestigatorLisaGennarini
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Trial DescriptionIsotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma - This partially randomized phase III trial studies isotretinoin with dinutuximab, aldesleukin, and sargramostim to see how well it works compared to isotretinoin alone following stem cell transplant in treating patients with neuroblastoma. Drugs used in chemotherapy, such as isotretinoin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Monoclonal antibodies, such as dinutuximab, may block tumor growth in different ways by targeting certain cells. Aldesleukin and sargramostim may stimulate a person's white blood cells to kill cancer cells. It is not yet known if chemotherapy is more effective with or without dinutuximab, aldesleukin, and sargramostim following stem cell transplant in treating neuroblastoma.NCT00026312
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Principal InvestigatorJonathanGill
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Trial DescriptionIvosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial) - This phase II Pediatric MATCH trial studies how well ivosidenib works in treating patients with solid tumors that have spread to other places in the body (advanced), lymphoma, or histiocytic disorders that have IDH1 genetic alterations (mutations). Ivosidenib may block the growth of cancer cells that have specific genetic changes in an important signaling pathway called the IDH pathway.NCT04195555
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Principal InvestigatorLisaGennarini
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Trial DescriptionLarotrectinib in Treating Patients With Previously Untreated TRK Fusion Solid Tumors and TRK Fusion Relapsed Acute Leukemia - This phase II trial studies the side effects and how well larotrectinib works in treating patients with previously untreated TRK fusion solid tumors and TRK fusion acute leukemia that has come back. Larotrectinib may stop the growth of cancer cells with TRK fusions by blocking the TRK enzymes needed for cell growth.NCT03834961
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Principal InvestigatorLisaGennarini
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Trial DescriptionLarotrectinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With NTRK Fusions (A Pediatric MATCH Treatment Trial) - This phase II Pediatric MATCH trial studies how well larotrectinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with NTRK fusions that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) and have come back (relapased) or does not respond to treatment (refractory). Larotrectinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.NCT03213704
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Principal InvestigatorLisaGennarini
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Trial DescriptionLight Treatment Effectiveness (LITE) Study - To compare the effectiveness, safety (tolerability), and duration of treatment response at 12 weeks of home versus office-based narrowband ultraviolet B phototherapy for the treatment of psoriasisNCT03726489
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Principal InvestigatorHollyKanavy
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Trial DescriptionLong-term Antipsychotic Pediatric Safety Trial - Main LAP01 study: The purpose is to evaluate the long-term pathologic weight changes associated with multi-year risperidone or aripiprazole therapy in 3 -NCT03522168
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Principal InvestigatorEricHollander
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Trial DescriptionLong-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies - This clinical trial keeps track of and collects follow-up information from patients who are currently enrolled on or have participated in a Children's Oncology Group study. Developing a way to keep track of patients who have participated in Children's Oncology Group studies may allow doctors learn more about the long-term effects of cancer treatment and help them reduce problems related to treatment and improve patient quality of life.NCT00736749
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Principal InvestigatorLisaGennarini
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Trial DescriptionLong-term Follow-up Study for Participants of Kite-Sponsored Interventional Studies Treated With Gene-Modified Cells - The goal of this clinical study is to learn more about the long-term safety, effectiveness and prolonged action of Kite study drugs, axicabtagene ciloleucel, brexucabtagene autoleucel, KITE-222, KITE-363, KITE-439, KITE-585, and KITE-718, in participants of Kite-sponsored interventional studies.NCT05041309
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Principal InvestigatorMendelGoldfinger
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Trial DescriptionLong-term Safety of Linaclotide in Pediatric Participants With FC or IBS-C - LIN-MD-66 is a Phase 3 open-label study with 24 weeks (Functional Constipation participants) or 52 weeks (Irritable bowel syndrome with constipation participants) of linaclotide exposure that will enroll pediatric participants (6-17 years of age) with FC or IBS-C who completed study intervention in studies LIN-MD-62, LIN-MD-63, orLIN-MD-64 based on the individual study criteria.NCT04166058
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Principal InvestigatorJohnThompson
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Trial DescriptionLong-Term Safety Study Of Tofacitinib In Patients With Juvenile Idiopathic Arthritis - Evaluate long-term safety and tolerability of tofacitinib in patients with JIA, who have previously participated in tofacitinib JIA studies.NCT01500551
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Principal InvestigatorDawnWahezi
Trial DescriptionLow-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma - This randomized phase II trial studies how well low-dose lenalidomide works compared with high-dose lenalidomide in treating younger patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas that have come back (recurrent), have not responded to treatment (refractory), or are growing, spreading, or getting worse (progressive). Lenalidomide is classified as an immunomodulatory drug as it boosts the immune system. It has other potential anti-tumor effects, for example, it may stop the growth of tumor cells by blocking blood flow to the tumor. It is not yet known whether low-dose lenalidomide is more or less effective than high-dose lenalidomide in treating patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas.NCT01553149
Disease/Condition -
Principal InvestigatorAdamLevy
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Trial DescriptionMail Order Mifepristone Study - This study aims to evaluate the feasibility, acceptability, and effectiveness of dispensing mifepristone via a mail-order pharmacy. The investigators will recruit patients at up to 10 primary care and internal medicines sites not currently providing abortion services around the country, as well as abortion clinic sites. Patients will go through routine in clinic visits to determine eligibility and then will receive the medications in the mail at their preferred address. The investigators will survey patients 3 and 14 days after the initial clinic visit and interview providers at the end of the study.NCT03913104
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Principal InvestigatorMarjiGold
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Trial DescriptionMaintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Patients With Newly Diagnosed Ependymoma - The primary aim of this randomized phase III trial was to study whether the addition of maintenance chemotherapy delivered after surgical resection and focal radiation would be better than surgery and focal radiation alone. The trial also studied if patients who received induction chemotherapy and then either achieved a complete response or went on to have a complete resection would also benefit from maintenance chemotherapy. Children ages 1-21 years with newly diagnosed intracranial ependymoma were included. There were 2 arms that were not randomized. One arm studied patients with Grade II tumors located in the supratentorial compartment that were completely resected. One arm studied patients with residual tumor and those patients all received maintenance chemotherapy after focal radiation. Chemotherapy drugs, such as vincristine sulfate, carboplatin, cyclophosphamide, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Radiation therapy uses high-energy x-rays to kill tumor cells. Specialized radiation therapy that delivers a high dose of radiation directly to the tumor may kill more tumor cells and cause less damage to normal tissue. Giving chemotherapy in combination with radiation therapy may kill more tumor cells and allow doctors to save the part of the body where the cancer started.NCT01096368
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Principal InvestigatorLisaGennarini
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Trial DescriptionMaster Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered T Cells in NY-ESO-1 and/or LAGE-1a Positive Solid Tumors - This trial will evaluate safety and efficacy of human engineered T-cell therapies, in participants with advanced tumors.NCT03967223
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Principal InvestigatorDavidLoeb
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Trial DescriptionMatched Targeted Therapy For High-Risk Leukemias and MDS - This research study is seeking to gain new knowledge about Recurrent, Refractory, or High Risk Leukemias in children and young adults. This study is evaluating the use of specialized testing called leukemia profiling. Once the profiling is performed, the results are evaluated by an expert panel of physicians, scientists and pharmacists. This may result in a recommendation for a specific cancer therapy or a clinical trial called matched targeted therapy (MTT). The results of the leukemia profiling and, if applicable, the MTT recommendation will be communicated to the participant's primary oncologist.NCT02670525
Disease/Condition -
Principal InvestigatorLisaGennarini
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Trial DescriptionMEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas - This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.NCT03962543
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Principal InvestigatorNagmaDalvi
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Trial DescriptionMetformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse - Primitive bone sarcoma are rare tumors with low options of therapy for patient treatment. OSTEOSARCOMA VERY POOR RESPONDER COHORT. Necrosis on primitive localized osteosarcoma represents one of the principal prognostic factors. Nowadays, for localized osteosarcoma there is no maintenance therapy that have shown to be effective. In ISG-OS1 study in patients with necrosis < 60% had an event free survival (EFS) at 3 yrs of 20% (Ferrari S ) in a more recent analysis (Tsuda Y 2020) patients with a necrosisNCT04758000
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Principal Investigator
Trial DescriptionMinimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease - This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).NCT03587272
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Principal InvestigatorMichelleLee
Trial DescriptionMismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome - This phase III trial compares hematopoietic (stem) cell transplantation (HCT) using mismatched related donors (haploidentical [haplo]) versus matched unrelated donors (MUD) in treating children, adolescents, and young adults with acute leukemia or myelodysplastic syndrome (MDS). HCT is considered standard of care treatment for patients with high-risk acute leukemia and MDS. In HCT, patients are given very high doses of chemotherapy or radiation therapy, which is intended to kill cancer cells that may be resistant to more standard doses of chemotherapy; unfortunately, this also destroys the normal cells in the bone marrow, including stem cells. After the treatment, patients must have a healthy supply of stem cells reintroduced or transplanted. The transplanted cells then reestablish the blood cell production process in the bone marrow. The healthy stem cells may come from the blood or bone marrow of a related or unrelated donor. If patients do not have a matched related donor, doctors do not know what the next best donor choice is or if a haplo related donor or MUD is better. This trial may help researchers understand whether a haplo related donor or a MUD HCT for children with acute leukemia or MDS is better or if there is no difference at all.NCT05457556
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Principal InvestigatorLisaGennarini
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Trial DescriptionMolecular and Genomic Profiling of Head and Neck Tumors - The purpose of this study is to study the genetic profile of head and neck tumors and their relationship to treatment response and outcomeNCT00200486
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Principal InvestigatorRichardSmith
Trial DescriptionMolecular-Guided Therapy for Childhood Cancer - The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup. This technology includes a genomic report based on DNA exomes and RNA sequencing that will be used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.NCT02162732
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Principal InvestigatorDanielWeiser
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Trial DescriptionMulti-Center African-American Inflammatory Bowel Disease Study (MAAIS) - The investigators are doing the research to discover genes that cause Inflammatory Bowel Disease (IBD) specifically in the African American population. African Americans with or without Crohn's disease or ulcerative colitis are eligible to join. If you agree to join the study, the investigators will ask for information about your health. The investigators will also ask you to give us a blood sample so that they may discover the genes that cause IBD. The blood sample may be collected at Johns Hopkins or any local facility convenient to you.NCT01169194
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Principal InvestigatorRubyGreywoode
Trial DescriptionMulticenter Prospective Cohort Study on Current Treatments of Legg-Calvé-Perthes Disease - Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0-14), but uncommon enough to have a sufficient number of patients from a single institution to perform a definitive prospective study comparing the results of current treatments. The present study will establish a database of prospectively identified patients with Legg-Calvé-Perthes (LCP) Disease and collect information regarding their presentation, treatment, and outcomes in the course of receiving currently available treatments. This study seeks to compare the outcomes of current treatments in the management of different age groups (ages 1-6, 6-8, 8-11, >11) of patients with Perthes disease at two- and five-year followup and at skeletal maturity. For each age group, two to three common treatment regimens currently used by practicing pediatric orthopaedic surgeons will be compared. The intervention a patient receives is determined through physician treatment expertise, and is not pre-determined by the study.NCT02040714
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Principal InvestigatorEricFornari
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Trial DescriptionNab-Paclitaxel and Gemcitabine for Recurrent/Refractory Sarcoma - The purpose of this study is to see if nab-paclitaxel combined with gemcitabine prevents the formation or growth of tumors in participants with relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma and other soft tissue sarcoma and to measure the length of time during and after treatment that their disease does not get worse. Researchers also want to find out if nab-paclitaxel combined with gemcitabine is safe and tolerable.NCT02945800
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Principal InvestigatorDanielWeiser
Trial DescriptionNational Cancer Institute "Cancer Moonshot Biobank" - This trial collects multiple tissue and blood samples, along with medical information, from cancer patients. The "Cancer Moonshot Biobank" is a longitudinal study. This means it collects and stores samples and information over time, throughout the course of a patient's cancer treatment. By looking at samples and information collected from the same people over time, researchers hope to better understand how cancer changes over time and over the course of medical treatments.NCT04314401
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Principal InvestigatorBalazsHalmos
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Trial DescriptionNational Collaborative to Improve Care of Children With Complex Congenital Heart Disease - The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, by improving the use of standards into everyday practice across pediatric cardiology centers, and by engaging parents as partners in the process.NCT02852031
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Principal InvestigatorNicoleSutton
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Trial DescriptionNatural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy - The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.NCT05112237
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Principal InvestigatorDaphneHsu
Trial DescriptionNCI COVID-19 in Cancer Patients, NCCAPS Study - This study collects blood samples, medical information, and medical images from patients who are being treated for cancer and have a positive test for SARS CoV-2, the new coronavirus that causes the disease called COVID-19. Collecting blood samples, medical information, and medical images may help researchers determine how COVID-19 affects the outcomes of patients undergoing cancer treatment and how having cancer affects COVID-19.NCT04387656
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Principal InvestigatorLisaGennarini
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Trial DescriptionNephrotic Syndrome Study Network - Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN biology, it is not surprising that the available therapies are imperfect. The therapies lack a clear biological basis, and as many families have experienced, they are often not beneficial, and in fact may be significantly toxic. Given these observations, it is essential that research be conducted that address these serious obstacles to effectively caring for patients. In response to a request for applications by the National Institutes of Health, Office of Rare Diseases (NIH, ORD) for the creation of Rare Disease Clinical Research Consortia, a number of affiliated universities joined together with The NephCure Foundation the NIDDK, the ORDR, and the University of Michigan in collaboration towards the establishment of a Nephrotic Syndrome (NS) Rare Diseases Clinical Research Consortium. Through this consortium the investigators hope to understand the fundamental biology of these rare diseases and aim to bank long-term observational data and corresponding biological specimens for researchers to access and further enrich.NCT01209000
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Principal InvestigatorFrederickKaskel
Trial DescriptionNeuroblastoma Maintenance Therapy Trial - Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.NCT02679144
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Principal InvestigatorDanielWeiser
Trial DescriptionNeurodevelopment of Hypoglycemic Neonates - The investigators propose to prospectively conduct a neurodevelopmental evaluation of SGA and late preterm neonates who underwent risk-based screening for hypoglycemia at newborn nursery during the first 24 hours of life based on AAP (American Academy of Pediatrics) hypoglycemia guidelines at 18 to 24 months of age. As per internal neonatal unit protocol (reflecting AAP guidelines), all neonates at risk of hypoglycemia (all preterm infants, term infants who are SGA or LGA and IDM) are routinely screened for hypoglycemia during the first 24 hours of life via bedside point of care glucose devices (see attached Weiler NICU (neonatal intensive care unit) hypoglycemia screening protocol). The investigators will compare neurodevelopmental outcomes of those who were and were not hypoglycemic in the newborn nursery based on electronic health record data.NCT04875624
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Principal InvestigatorThomasHavranek
Trial DescriptionNeuropsychological and Behavioral Testing in Younger Patients With Cancer - This research trial studies neuropsychological (learning, remembering or thinking) and behavioral outcomes in children and adolescents with cancer by collecting information over time from a series of tests.NCT00772200
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Principal InvestigatorLisaGennarini
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Trial DescriptionNeuropsychological, Social, Emotional, and Behavioral Outcomes in Children With Cancer NCT00772200
Disease/ConditionChildhood Malignant Neoplasm
Principal InvestigatorLisa Gennarini
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Trial DescriptionNivolumab or Nivolumab and Azacitidine in Patients With Recurrent, Resectable Osteosarcoma - The purpose of this study is to evaluate the safety and efficacy of nivolumab, or nivolumab in combination with azacitidine in participants with recurrent, resectable osteosarcomaNCT03628209
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Principal InvestigatorDanielWeiser
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry - Continuation of the CARRA Registry as described in the protocol will support data collection on patients with pediatric-onset rheumatic diseases. The CARRA Registry will form the basis for future CARRA studies. In particular, this observational registry will be used to answer pressing questions about therapeutics used to treat pediatric rheumatic diseases, including safety questions.NCT02418442
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Principal InvestigatorDawnWahezi
Trial DescriptionONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study) - This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with ONC201 following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.NCT05580562
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Principal InvestigatorAdiliaHormigo
Trial DescriptionOptimize PRO Study - The purpose of this study is to collect clinical evidence on valve performance and procedural outcomes associated with an "optimized" TAVR care pathway and post-TAVR conduction disturbance pathway while using the Evolutâ„¢ PRO and Evolutâ„¢ PRO+ devices. The purpose of the addendum is to collect post-market clinical evidence on valve performance and procedural outcomes associated with the Evolut FX Device.NCT04091048
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Principal InvestigatorMohamedAzeemLatib
Trial DescriptionOutcomes of Neonatal Acute Kidney Injury In Premature Infants - The objective of this protocol is to investigate the impact of prematurity, with or without associated acute kidney injury (AKI), on the future risk of chronic kidney disease (CKD) by establishing a patient registry and biorepository. Serum and urine samples will be collected serially from premature infants admitted to the neonatal intensive care unit (NICU) at Albert Einstein College of Medicine/Weiler Hospital and subsequently followed in the NICU follow-up and pediatric nephrology ambulatory subspecialty clinics. The biorepository will be linked to a comprehensive clinical database.NCT02375854
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Principal InvestigatorMamtaFuloria
Trial DescriptionOxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD - This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.NCT04930445
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Principal InvestigatorHennyBillett
Trial DescriptionPathogen Identification in Pediatric Hematopoietic Stem Cell Transplant Patients With Suspected Lower Respiratory Tract Infection - This is a multicenter prospective collection of leftover respiratory tract secretions, paired blood and NP swabs, and clinical circumstances from pediatric HCT patients, followed by next generation genomic sequencing, transcriptome analysis, protein biomarker measurement, and statistical modeling.NCT02926612
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Principal InvestigatorDeepaManwani
Trial DescriptionPediatric Lupus Trial of Belimumab Plus Background Standard Therapy - This is a multi-center study to evaluate the safety, pharmacokinetics, and efficacy of belimumab intravenous (IV) in pediatric patients 5 to 17 years of age with active systemic lupus erythematosusNCT01649765
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Principal InvestigatorDawnWahezi
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Trial DescriptionPediatric Teduglutide Registry - Pediatric specific post-marketing registry to evaluate the long-term safety and efficacy of teduglutide.NCT04832087
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Principal InvestigatorJohnThompson
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Trial DescriptionPhase 2/3 Adaptive Study of VX-147 in Adults and Adolescents With APOL1- Mediated Proteinuric Kidney Disease - The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.NCT05312879
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Principal InvestigatorMichaelRoss
Trial DescriptionPictographs for Preventing Wrong-Patient Errors in NICUs - Newborns in the neonatal intensive care unit (NICU) are at high risk for wrong-patient errors. Effective 2019, The Joint Commission requires that health systems adopt distinct methods of newborn identification as part of its National Patient Safety Goals. Displaying patient photographs in the electronic health record (EHR) is a promising strategy to improve identification of children and adults, but is unlikely to be effective for identifying newborns. This study assesses the use of Pictographs as a "photo equivalent" for improving identification of newborns in the NICU. This multi-site, two-arm, parallel group, cluster randomized controlled trial will test the effectiveness of Pictographs for preventing wrong-patient order errors in the NICU. Pictographs consist of three elements: 1) pictorial symbols of easy-to-remember objects (e.g., rainbow, lion); 2) the infant's given name (when available); and 3) a color-coded border indicating the infant's sex. The study will be conducted at three academic medical centers that utilize Epic EHR. All parents or guardians will be asked to select a unique Pictograph for each infant admitted to the NICU to be displayed on the isolette and in the EHR for the duration of the infant's hospital stay. All clinicians with the authority to place electronic orders in the study NICUs will be randomly assigned to either the intervention arm (Pictographs displayed in the EHR) or the control arm (no Pictographs displayed in the EHR). The main hypothesis is that clinicians assigned to view Pictographs in the EHR will have a significantly lower rate of wrong-patient order errors in the NICU versus clinicians assigned to no Pictographs. The primary outcome is wrong-patient order sessions, defined as a series of orders placed for a single patient by a single clinician that contains at least one wrong-patient order. The Wrong-Patient Retract-and-Reorder (RAR) measure, a validated, reliable, and automated method for identifying wrong-patient orders, will be used as the primary outcome measure. The Wrong-Patient RAR measure identifies one or more orders placed for a patient that are retracted within 10 minutes, and then reordered by the same clinician for a different patient within the next 10 minutes. In the validation study conducted at a large academic medical center, real-time telephone interviews with clinicians confirmed that 76.2% of RAR events were correctly identified by the measure as wrong-patient orders.NCT03960099
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Principal InvestigatorKatieForman
Trial DescriptionPrevalence of Carbohydrate Intolerance in Lean and Obese Children - The prevalence of obesity in children is reaching epidemic proportions. Excess adiposity is more than just a cosmetic problem, having substantial metabolic consequences. Insulin resistance, hyperinsulinemia, impaired glucose tolerance, and frank diabetes are often seen in obese children. In this study the prevalence of impaired glucose (carbohydrate) tolerance in lean children with a family history of diabetes and obese children with acanthosis nigricans with or without a family history of diabetes mellitus will be studied.NCT00000112
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Principal InvestigatorMarioGarcia
Trial DescriptionPreventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission - The purpose of this research study is to evaluate a new investigational drug to prevent reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The objectives of this study will be to monitor for safety and look at efficacy of DFMO. The safety of the proposed dosing regimen in this trial will be tested by an on-going risk/benefit assessment during the study. A patient benefiting from treatment, not progressing on therapy, and in the absence of any safety issues associated with DFMO may continue on treatment up to 27 cycles with the expectation that there will be an overall clinical benefit. The procedures involved in this study include Medical history, Physical exam, Vital signs (blood pressure, pulse, temperature), Blood tests, Urine tests, MRI or CT scan of the tumor(s), meta-iodobenzylguanidine (MIBG) scans, and Bone marrow aspirations. All of these tests and procedures are considered standard of care for this population. Drug administration is also part of this protocol, including an investigational new drug called DFMO. The proposed dosing regimen is an oral dose of DFMO tablets two times a day for each day while on study. There will be 27 cycles. Each cycle will be 28 days in length.NCT02395666
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Principal InvestigatorDanielWeiser
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Trial DescriptionPreventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA) - This is a research study to test whether a once-weekly injection of abatacept will prevent the progression of Juvenile Idiopathic Arthritis (JIA) to a more severe form. To evaluate the effectiveness of a 24-week course of treatment with abatacept plus usual care versus usual care to prevent polyarthritis (≥5 joints), uveitis, or treatment with other systemic medication within 18 months of randomization in children with recent-onset limited JIA.NCT03841357
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Principal InvestigatorDawnWahezi
Trial DescriptionProject: Every Child for Younger Patients With Cancer - This study gathers health information for the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.NCT02402244
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Principal InvestigatorLisaGennarini
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Trial DescriptionPromoting Asthma Guidelines and Management Through Technology-Based Intervention and Care Coordination - The overall goal of this research study is to test the effectiveness of a multifaceted and multi-level prompting intervention in a real world urban primary care office setting on improving provider-delivered guideline-based asthma care and reducing asthma morbidity among urban children with persistent or uncontrolled asthma.NCT03066596
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Principal InvestigatorMarinaReznik
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Trial DescriptionPROSpect: Prone and Oscillation Pediatric Clinical Trial - Severe pediatric acute respiratory distress syndrome (PARDS) is a life-threatening and frequent problem experienced by thousands of children each year. Little evidence supports current supportive practices during their critical illness. The overall objective of this study is to identify the best positional and/or ventilation practice that leads to improved patient outcomes in these critically ill children. We hypothesize that children with high moderate-severe PARDS treated with either prone positioning or high-frequency oscillatory ventilation (HFOV) will demonstrate more days off the ventilator when compared to children treated with supine positioning or conventional mechanical ventilation (CMV).NCT03896763
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Principal InvestigatorSaraSoshnick
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Trial DescriptionRadiation Therapy With or Without Combination Chemotherapy or Pazopanib Before Surgery in Treating Patients With Newly Diagnosed Non-rhabdomyosarcoma Soft Tissue Sarcomas That Can Be Removed by Surgery - This randomized phase II/III trial studies how well pazopanib, when combined with chemotherapy and radiation therapy or radiation therapy alone, work in the treatment of patients with newly diagnosed non-rhabdomyosarcoma soft tissue sarcomas that can eventually be removed by surgery. Radiation therapy uses high energy x-rays to kill tumor cells. Drugs used in chemotherapy, such as ifosfamide and doxorubicin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Pazopanib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether these therapies can be safely combined and if they work better when given together in treating patients with non-rhabdomyosarcoma soft tissue sarcomas.NCT02180867
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Principal InvestigatorLisaGennarini
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Trial DescriptionRandomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants - The overall goal of this study is to determine the clinical benefit and safety of antiviral therapy for asymptomatic congenital cytomegalovirus (cCMV) infected hearing-impaired infants. We will conduct a multi-center double-blind randomized placebo-controlled trial to determine whether hearing-impaired infants with asymptomatic cCMV have better hearing and language outcomes if they receive valganciclovir antiviral treatment. We will also determine the safety of antiviral valganciclovir therapy for asymptomatic cCMV-infected hearing impaired infants. This study will be unique in that the cohort enrolled will only include hearing-impaired infants with asymptomatic cCMV. Primary Objective: To determine if treatment of cCMV-infected hearing impaired infants with isolated hearing loss with the antiviral drug valganciclovir reduces the mean slope of total hearing thresholds over the 20 months after randomization compared to untreated cCMV-infected infants with isolated hearing loss. Main Secondary Objectives: To determine if valganciclovir treatment improves the following outcomes when compared to the control group: The slope of best ear hearing thresholds over the 20 months after randomization. The MacArthur-Bates Communicative Development Inventory (CDI) percentile score for words produced at 20 months of age. To evaluate safety measures based on all grade 3 or greater new adverse events designated by the NIAID Division of AIDS (DAIDS) toxicity tables.NCT03107871
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Principal InvestigatorMichelNassar
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Trial DescriptionReduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma - This phase II trial studies how well reduced doses of radiation therapy to the brain and spine (craniospinal) and chemotherapy work in treating patients with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma. Recent studies using chemotherapy and radiation therapy have been shown to be effective in treating patients with WNT-driven medulloblastoma. However, there is a concern about the late side effects of treatment, such as learning difficulties, lower amounts of hormones, or other problems in performing daily activities. Radiotherapy uses high-energy radiation from x-rays to kill cancer cells and shrink tumors. Drugs used in chemotherapy, such as cisplatin, vincristine sulfate, cyclophosphamide and lomustine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving reduced craniospinal radiation therapy and chemotherapy may kill tumor cells and may also reduce the late side effects of treatment.NCT02724579
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Principal InvestigatorLisaGennarini
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Trial DescriptionReduced Intensity Haploidentical BMT for High Risk Solid Tumors - The purpose of this study is to see if giving reduced intensity chemotherapy, haploidentical bone marrow, post-transplant cyclophosphamide and shortened duration tacrolimus is safe and feasible for patients with very high-risk solid tumors.NCT01804634
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Principal InvestigatorDavidLoeb
Trial DescriptionRegistry for Participants With Short Bowel Syndrome - This is a global prospective, observational, multi-center registry to evaluate the long-term safety profile for participants with short bowel syndrome (SBS) who are treated with teduglutide in a routine clinical setting. The registry will also evaluate the long-term clinical outcomes in participants with SBS. SBS participants treated and not treated with teduglutide will be enrolled.NCT01990040
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Principal InvestigatorJohnThompson
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Trial DescriptionRegistry of Asthma Patients Initiating DUPIXENT® - The primary objective of the study is to characterize the patients who initiate treatment for asthma with DUPIXENT® in a real-world setting to understand the attributes of treated patients in real life. This includes characterization of: Patient demographics (eg, gender, age, and race) Patient baseline characteristics (eg, prior medications and procedures, medical history, asthma history, weight, height) The secondary objectives of the study are: To characterize real-world use patterns of DUPIXENT® for asthma To assess the long-term effectiveness of DUPIXENT® in asthma patients in a real-world setting To assess effectiveness on comorbid type 2 inflammatory conditions in asthma patients treated with DUPIXENT® To collect long-term safety data on study participants in the real-world settingNCT04287621
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Principal InvestigatorGoldaHudes
Trial DescriptionRegistry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) - Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases. Until recently, the mainstay of treatment for these patients was supportive medical care. However, advances in medical treatment focusing on gene replacement, gene enhancement, motor neuron protection and muscle enhancement is likely to change the management and prognosis of these patients in the future. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options.NCT04174157
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Principal InvestigatorLESLIEDELFINER
Trial DescriptionRenal Tumors Classification, Biology, and Banking Study - PRIMARY OBJECTIVES: I. Classify patients with renal tumors by histological categorization, surgico-pathological stage, presence of metastases, age at diagnosis, tumor weight, and loss of heterozygosity for chromosomes 1p and 16q, to define eligibility for a series of therapeutic studies. (As of Amendment 8, this aim is limited to patients with institutionally classified Stage I to IV focal or diffuse anaplasia identified at initial biopsy or primary nephrectomy OR at delayed nephrectomy/second biopsy.) II. To maintain a biological samples bank to make specimens available to scientists to evaluate additional potential biological prognostic variables and for the conduct of other research by scientists. SECONDARY OBJECTIVES: I. To monitor outcome for those patients who are not eligible for a subsequent therapeutic study. II. To describe whether the pulmonary tumor burden correlates with outcome in Stage IV patients.(Completed as of Amendment 7) III. To describe the sensitivity and specificity of abdominal computed tomography (CT) by comparison with surgical and pathologic findings for identification of local tumor spread beyond the renal capsule to adjacent muscle and organs, lymph node involvement at the renal hilum and in the retroperitoneum, preoperative tumor rupture and metastases to the liver. (Completed as of Amendment 7) IV. To compare the sensitivity and specificity of preoperative abdominal CT and MRI for the identification and differentiation of nephrogenic rests and Wilms tumor in children with multiple renal lesions. (Completed as of Amendment 7) V. To correlate the method of conception (natural versus assisted reproductive technology) with the development of Wilms tumor. (Completed as of Amendment 7) VI. To evaluate the frequency of integrase interactor 1 (INI1) mutations in renal and extrarenal malignant rhabdoid tumor of the kidney and to determine the incidence of germline and inherited versus somatic mutations to facilitate clinical correlations on the companion study AREN0321. (Completed as of Amendment 7) OUTLINE: Tumor tissue, blood, and urine samples are collected for research studies, including immunohistochemistry. CT scans and MRIs are also performed. Loss of heterozygosity analyses (chromosome 1p and 16q) are performed by extraction of DNA. DNA polymorphisms are assayed by polymerase chain reaction using standard methodology. Leftover specimens are archived for future studies. (LOH and INI1 testing discontinued as of April 2014) Patients are followed up periodically for 5 years.NCT00898365
Disease/ConditionAdult Cystic Nephroma
Anaplastic Kidney Wilms Tumor
Angiolipoma
Cellular Congenital Mesoblastic Nephroma
Classic Congenital Mesoblastic Nephroma
Clear Cell Sarcoma of the Kidney
Congenital Mesoblastic Nephroma
Cystic Partially Differentiated Kidney Nephroblastoma
Diffuse Hyperplastic Perilobar Nephroblastomatosis
Extrarenal Rhabdoid Tumor
Kidney Medullary Carcinoma
Kidney Neoplasm
Kidney Oncocytoma
Kidney Wilms Tumor
Metanephric Adenofibroma
Metanephric Adenoma
Metanephric Stromal Tumor
Metanephric Tumor
Mixed Congenital Mesoblastic Nephroma
Ossifying Renal Tumor of Infancy
Papillary Renal Cell Carcinoma
Renal Cell Carcinoma
Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions
Rhabdoid Tumor of the Kidney
Wilms Tumor
Principal InvestigatorLisa Gennarini
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Trial DescriptionREpeated ASSEssment of SurvivorS in Intracerebral Hemorrhage Study - The investigators propose to perform serial detailed cognitive, motor, behavioral, and blood collection follow-up using longitudinal structured telephone interviews of an anticipated 350 ICH survivors enrolled in Minimally Invasive Surgery Plus Alteplase for Intracerebral Hemorrhage Evacuation (MISTIE) III and ENRICH trials to identify specific cognitive and motor impairment and to perform RNA sequencing to evaluate for evidence of chronic inflammation. The investigators' expected sample size in 2022 accounts for mortality attrition of 10%/year.NCT05611918
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Principal InvestigatorRishiMalhotra
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Trial DescriptionReproductive Health Program in Patients With Cancer - This clinical trial studies reproductive health program in patients with cancer. A reproductive health program may improve patients' understanding of reproductive risks and receipt of appropriate treatment to achieve their reproductive health goals.NCT01806129
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Principal InvestigatorDellaMakower
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Trial DescriptionResponse and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma - This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.NCT02176967
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Principal InvestigatorLisaGennarini
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Trial DescriptionResponse-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome - This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.NCT02521493
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Principal InvestigatorLisaGennarini
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Trial DescriptionRisk of Wrong-Patient Errors With Multiple Records Open - Currently, at least 70,000 U.S. physicians use computerized provider order entry (CPOE) to place orders. This number is expected to rise sharply as hospitals continue to take advantage of federal incentives and adopt electronic health record (EHR) technology. Although CPOE is associated with a reduction in medical errors, when orders are placed electronically certain types of errors, including placing orders on the wrong patient, may occur more frequently. The mechanism by which multiple patient records opened simultaneously can lead to wrong-patient errors may be related to the ease with which users can toggle between patient records and the similar looking computer screens. The magnitude of this risk needs to be established to help Health Information Technology (IT) leadership decide how to safely implement CPOE systems. There have been no studies demonstrating whether multiple records increase the risk of wrong-patient errors, by how much, and if any increase is dependent on the number of records open. This research project is an important first step in quantifying this risk.This will be the first study to achieve the following aims: Assess the relationship between the number of records open at the time of placing an order and the risk of placing an order on the wrong patient. Compare the incidence of wrong-patient orders in a "restricted environment" that limits providers to only one record open at a time to an "unrestricted environment" where users can open a maximum of four records at once. Use results to inform a larger-scale health IT implementation research project evaluating the balance between the wrong-patient error risks and potential efficiency gains of having multiple records open at once, with rigorous research methodologies. Disseminate results to help inform decisions on how to safely implement EHR systems.NCT02876588
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Principal InvestigatorWilliamSouthern
Trial DescriptionRisk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma - This partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute lymphoblastic leukemia or B-lineage lymphoblastic lymphoma that is found only in the tissue or organ where it began (localized). Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy), giving the drugs in different doses, and giving the drugs in different combinations may kill more cancer cells.NCT01190930
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Principal InvestigatorLisaGennarini
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Trial DescriptionRisk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer - This phase III trial studies the side effects and how well risk-based therapy works in treating younger patients with newly diagnosed liver cancer. Surgery, chemotherapy drugs (cancer fighting medicines), and when necessary, liver transplant, are the main current treatments for hepatoblastoma. The stage of the cancer is one factor used to decide the best treatment. Treating patients according to the risk group they are in may help get rid of the cancer, keep it from coming back, and decrease the side effects of chemotherapy.NCT00980460
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Principal InvestigatorLisaGennarini
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Trial DescriptionRSSearch Patient Registry-Long Term Study of Use of SRS/SBRT - The RSSearch® Registry is an international multi-year database designed to track SRS/SBRT (Stereotactic Radiosurgery/Stereotactic Body Radiation Therapy) utilization, treatment practices and outcomes to help determine, over time, the most effective use of these systems in management of patients with life threatening tumors and other diseases. (This study was formally called ReCKord and included the CyberKnife only; The ReCKord study continues as a CyberKnife subset of RSSearch.)NCT01885299
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Principal InvestigatorMadhurGarg
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Trial DescriptionSafety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children - This will be an open-label, randomized, multicenter, efficacy and safety study of weekly MOD-4023 compared to daily Genotropin therapy in pre-pubertal children with growth hormone deficiency.NCT02968004
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Principal InvestigatorPingZhou
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Trial DescriptionSafety and Pharmacokinetics of Ceftolozane/Tazobactam in Pediatric Participants With Nosocomial Pneumonia (MK-7625A-036) - This is a phase 1, open-label, non-comparative, multicenter clinical study to evaluate the safety, tolerability, and pharmacokinetics of ceftolozane/tazobactam (MK-7625A) in pediatric participants with nosocomial pneumonia (NP).NCT04223752
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Principal InvestigatorMichelleCollins-Ogle
Trial DescriptionSafety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation - This study will evaluate the safety of infusion of the investigational cord blood units by carefully documenting all infusion-related problems.NCT01656603
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Principal InvestigatorMichelleLee
Trial DescriptionSamotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial) - This phase II Pediatric MATCH trial studies how well samotolisib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with TSC or PI3K/MTOR mutations that have spread to other places in the body (metastatic) and have come back (recurrent) or do not respond to treatment (refractory). Samotolisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.NCT03213678
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Principal InvestigatorLisaGennarini
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Trial DescriptionSC-PEG Asparaginase vs. Oncaspar in Pediatric Acute Lymphoblastic Leukemia (ALL) and Lymphoblastic Lymphoma - This study is being conducted to learn about the effects of SC-PEG, which is a new form of a chemotherapy drug called asparaginase. Asparaginase is used to treat ALL and lymphoblastic lymphoma. The standard form of asparaginase, called Elspar, is given in the muscle once a week for 30 weeks. There are other forms of asparaginase. The investigators will be studying two of these: Oncaspar and Calaspargase Pegol (SC-PEG). The investigators have previously studied giving Oncaspar in the vein (instead of the muscle) every 2 weeks in patients with ALL, and have shown that this dosing did not lead to any more side effects than Elspar given weekly in the muscle. The study drug, SC-PEG, is very similar but not identical to Oncaspar. SC-PEG has been given in the vein to children and adolescents with ALL as part of other research studies, and it appears to last longer in the blood after a dose than Oncaspar. It has not yet been approved by the FDA. The goal of this research study is to learn whether the side effects and drug levels of SC-PEG given in the vein every 3 weeks are similar to Oncaspar given into the vein about every 2 weeks. The study will also help to determine whether changing treatment for children and adolescents with ALL with high levels of minimal residual disease may improve cure rates. Measuring minimal disease (MRD) is a laboratory test that finds low levels of leukemia cells that the investigators cannot see under the microscope. In the past, it has been shown that children and adolescents with ALL with high levels of MRD after one month of treatment are less likely to be cured than those with low levels of MRD. Therefore, on the study, the bone marrow and blood at the end of the first month of treatment will be measured in participants with leukemia, and changes in therapy will be implemented based on this measurement. It is not known for sure that changing treatment will improve cure rates. MRD levels can only be measured if the marrow is filled with cancer cells at the time of diagnosis. Therefore, MRD studies will only be done in children and adolescents with ALL and not in those with lymphoblastic lymphoma. Another part of the study is to determine whether giving antibiotics during the first month of treatment even to participants without fever will prevent serious infections in the blood and other parts of the body. About 25% of children and adolescents with ALL and lymphoblastic lymphoma who receive standard treatment develop a serious blood infection from a bacteria during the first month of treatment. Typically, antibiotics (medicines that fight bacteria) are given by vein only after a child with leukemia or lymphoma develops a fever or have other signs of infection. In this study, antibiotics will be given by mouth or in the vein to all participants during the first month of treatment, whether or not they develop fever. Another goal of the study to learn how vitamin D levels relate to bone problems (such as broken bones or fractures) that children and adolescents with ALL and lymphoblastic lymphoma experience while on treatment. Some of the chemotherapy drugs used to treat ALL and lymphoblastic lymphoma can make bones weaker, which make fractures more likely. Vitamin D is a natural substance from food and sunlight that can help keep bones strong. The investigators will study how often participants have low levels of vitamin D while receiving chemotherapy, and, for those with low levels, whether giving vitamin D supplements will increase those levels. Another focus of the study is to learn more about the biology of ALL and lymphoblastic lymphoma by doing research on blood, bone and spinal fluid bone marrow samples. The goal of this research is to improve treatment for children with leukemia in the future.NCT01574274
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Principal InvestigatorLisaGennarini
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Trial DescriptionScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program - ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a panel of additional conditions. The study has three primary objectives: 1) define the analytic and clinical validity of multi-tiered screening assays for a flexible panel of disorders, 2) determine disease incidence in an ethnically diverse population, and 3) assess the impact of early diagnosis on health outcomes. Over a five-year period, ScreenPlus aims to screen 175,000 infants born in nine high birthrate, ethnically diverse pilot hospitals in New York for a flexible panel of 14 rare genetic disorders. This study will also involve an evaluation of the Ethical, Legal and Social issues pertaining to NBS for complex disorders, which will be done via online surveys that will be directed towards ScreenPlus parents who opt to participate and qualitative interviews with families of infants who are identified through ScreenPlus.NCT05368038
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Principal InvestigatorSuhasNafday
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Trial DescriptionSelpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial - This phase II pediatric MATCH treatment trial studies how well selpercatinib works in treating patients with solid tumors that may have spread from where they first started to nearby tissue, lymph nodes, or distant parts of the body (advanced), lymphomas, or histiocytic disorders that have activating RET gene alterations. Selpercatinib may block the growth of cancer cells that have specific genetic changes in an important signaling pathway (called the RET pathway) and may reduce tumor size.NCT04320888
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Principal InvestigatorLisaGennarini
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Trial DescriptionSickle Cell Disease Biofluid Chip Technology (SCD BioChip) - 'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949. The pathophysiology of SCD is a consequence of abnormal polymerization of sickle hemoglobin (HbS) and its effects on red cell membrane properties, shape, and density, and subsequent critical changes in inflammatory cell and endothelial cell function. Our goal is to understand the impact of CMA abnormalities in SCD, by interrogating a number of recognized interactions in a range of clinical phenotypes. To date, correlative studies in SCD, by us and others, have range between clinical reports, based on tests, interventions, and chart review of individuals or groups of individuals and, at the other extreme, identification of functional gene polymorphisms based on population studies. The investigators wish to augment these studies through a systematic examination of cellular membrane properties and activation status. Of hematologic disorders, SCD may be unusually susceptible to such an examination.NCT02824471
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Principal InvestigatorDeepaManwani
Trial DescriptionSimple Bone Cysts in Kids - Simple bone cysts (SBCs) are cysts filled with fluid that occur most frequently in the long bones (arms or legs) of children. There are many ways to treat SBCs but it is unclear if one is better than another. The purpose of this research trial is to compare the effectiveness of two common treatments that are used by surgeons today.NCT02193841
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Principal InvestigatorJacobSchulz
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Trial DescriptionStaged Complete Revascularization for Coronary Artery Disease vs Medical Management Alone in Patients With AS Undergoing Transcatheter Aortic Valve Replacement - Patients undergoing transcatheter aortic valve replacement (TAVR) often have concomitant coronary artery disease (CAD) which may adversely affect prognosis. There is uncertainty about the benefits and the optimal timing of revascularization for such patients. There is currently clinical equipoise regarding the management of concomitant CAD in patients undergoing TAVR. Some centers perform routine revascularization with percutaneous coronary intervention (PCI) (either before or after TAVR), while others follow an alternative strategy of medical management. The potential benefits and optimal timing of PCI in these patients are unknown. As TAVR expands to lower risk patients, and potentially becomes the preferred therapy for the majority of patients with severe aortic stenosis, the optimal management of concomitant coronary artery disease will be of increasing importance. The COMPLETE TAVR study will determine whether, on a background of guideline-directed medical therapy, a strategy of complete revascularization involving staged PCI using drug eluting stents to treat all suitable coronary artery lesions is superior to a strategy of medical therapy alone in reducing the composite outcome of Cardiovascular Death, new Myocardial Infarction, Ischemia-driven Revascularization or Hospitalization for Unstable Angina or Heart Failure. The study will be a randomized, multicenter, open-label trial with blinded adjudication of outcomes. Patients will be screened and consented for elective transfemoral TAVR and randomized within 96 hours of successful balloon expandable TAVR. Complete Revascularization: Staged PCI using third generation drug eluting stents to treat all suitable coronary artery lesions in vessels that are at least 2.5 mm in diameter and that are amenable to treatment with PCI and have a ≥70% visual angiographic diameter stenosis. Staged PCI can occur any time from 1 to 45 days post successful transfemoral TAVR. Vs. Medical Therapy Alone: No further revascularization of coronary artery lesions. All patients, regardless of randomized treatment allocation, will receive guideline-directed medical therapy consisting of risk factor modification and use of evidence-based therapies. The COMPLETE TAVR study will help address the current lack of evidence in this area. It will likely impact both the global delivery of health care and the management and clinical outcomes of all patients undergoing TAVR with concomitant CAD.NCT04634240
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Principal InvestigatorMarkMenegus
Trial DescriptionStandard-Dose Combination Chemotherapy or High-Dose Combination Chemotherapy and Stem Cell Transplant in Treating Patients With Relapsed or Refractory Germ Cell Tumors - This randomized phase III trial studies how well standard-dose combination chemotherapy works compared to high-dose combination chemotherapy and stem cell transplant in treating patients with germ cell tumors that have returned after a period of improvement or did not respond to treatment. Drugs used in chemotherapy, such as paclitaxel, ifosfamide, cisplatin, carboplatin, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as filgrastim or pegfilgrastim, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. It is not yet known whether high-dose combination chemotherapy and stem cell transplant are more effective than standard-dose combination chemotherapy in treating patients with refractory or relapsed germ cell tumors.NCT02375204
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Principal InvestigatorBenjaminGartrell
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Trial DescriptionStopping Tyrosine Kinase Inhibitors in Affecting Treatment-Free Remission in Patients With Chronic Phase Chronic Myeloid Leukemia - This phase II trial studies how stopping tyrosine kinase inhibitors will affect treatment-free remission in patients with chronic myeloid leukemia in chronic phase. When the level of disease is very low, it's called molecular remission. TKIs are a type of medication that help keep this level low. However, after being in molecular remission for a specific amount of time, it may not be necessary to take tyrosine kinase inhibitors. It is not yet known whether stopping tyrosine kinase inhibitors will help patients with chronic myeloid leukemia in chronic phase continue or re-achieve molecular remission.NCT03817398
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Principal InvestigatorLisaGennarini
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Trial DescriptionStudy Assessing CLENPIQ as Bowel Preparation for Pediatric Colonoscopy - Bowel preparation for pediatric colonoscopy.NCT04113382
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Principal InvestigatorInnaNovak
Trial DescriptionStudy Evaluating Brexucabtagene Autoleucel (KTE-X19) in Pediatric and Adolescent Participants With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma - The primary objectives of this study are to evaluate the safety and efficacy of brexucabtagene autoleucel (KTE-X19) in pediatric and adolescent participants with relapsed/refractory (r/r) B-precursor acute lymphoblastic leukemia (ALL) or relapsed or refractory (r/r) B-cell non-Hodgkin lymphoma (NHL). As of October 2022, no further patients with acute B-cell Acute Lymphoblastic Leukemia (ALL) will be asked to join the study. The study remains open for recruitment for patients that have B-cell Non Hodgkin Lymphoma (NHL).NCT02625480
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Principal InvestigatorEllenFraint
Trial DescriptionStudy Evaluating Subjects With Distal Renal Tubular Acidosis - This is a phase 3, prospective, multicenter, randomized, double-blinded, placebo-controlled withdrawal study comparing the efficacy of ADV7103 versus placebo in preventing the development of metabolic acidosis defined by serum bicarbonate levels in pediatric (6 months to < 18 years of age) and adult (18 to 65 years of age) subjects with primary Distal Renal Tubular Acidosis (dRTA).NCT03644706
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Principal InvestigatorKimberlyReidy
Trial DescriptionStudy for the Prediction of Active Rejection in Organs Using Donor-derived Cell-free DNA Detection - This is a multi-center prospective study designed to collect blood samples from transplant patients in order to improve Natera's method for determining allograft rejection status using the donor-derived cell-free DNA analysis, called Prospera.NCT03984747
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Principal InvestigatorNicoleHayde
Trial DescriptionStudy of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients - The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months toNCT03474965
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Principal InvestigatorDeepaManwani
Trial DescriptionStudy of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant - This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).NCT03301168
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Principal InvestigatorMichelleLee
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Trial DescriptionStudy of Kidney Tumors in Younger Patients - This research trial studies kidney tumors in younger patients. Collecting and storing samples of tumor tissue, blood, and urine from patients with cancer to study in the laboratory may help doctors learn more about changes that occur in deoxyribonucleic acid (DNA) and identify biomarkers related to cancer.NCT00898365
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Principal InvestigatorLisaGennarini
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Trial DescriptionStudy of Mycobacterial Infections - This study will examine the symptoms, course of disease and treatment of non-tuberculous mycobacterial (NTM) infections, as well as the genetics involved in these infections. Patients with NTM have recurrent lung infections and sometimes infections of the skin and other organs as well. They may also have curvature of the spine, barrel chest, and heart valve weakness. The study will compare the features of NTM with those of Job syndrome and cystic fibrosis, other diseases involving recurrent infections of the lungs and possibly other organs. Patients with diagnosed or suspected non-tuberculous mycobacterial infection, cystic fibrosis or Job syndrome may be eligible for this study. All participants will have a medical and family history, blood and urine tests, imaging studies that may include X-rays, computed tomography (CT) or magnetic resonance imaging (MRI) scans, and DNA and other genetic studies. In addition, all patients with Job syndrome and cystic fibrosis, and patients with NTM who have lung disease undergo the following procedures: Scoliosis survey X-rays of the spine to look for curvature or other abnormalities of the spinal column Echocardiography imaging test that uses sound waves to examine the heart chambers and valves Electrocardiogram measurement of the electrical activity of the heart Pulmonary function tests breathing tests to measure how much air the patient can move into and out of the lungs Body measurements measurements of height, weight, arm span, finger length, etc. Joint function assessment of joint mobility using different maneuvers to test flexibility of joints and ligaments Examination of physical features that might be associated with NTM, such as high arched palate of the mouth, flat feet, or certain skin features Dermatology (skin) examination for reactive skin conditions or other skin problems and possibly a skin biopsy (surgical removal of a small skin tissue sample for microscopic examination) Interview with genetics specialist These tests may require several days to complete. Patients with NTM will also be examined by a cystic fibrosis specialist and may have a sweat test. In addition, NTM patients will be asked to return to NIH every year for 5 years for follow-up tests, if medically indicated, including CT of the chest, scoliosis survey and examination by other specialists.NCT00018044
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Principal InvestigatorStevenPorcelli
Trial DescriptionStudy Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors - A study to learn about safety and find out maximum tolerable dose of palbociclib given in combination with chemotherapy (temozolomide with irinotecan or topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors (phase 1). Neuroblastoma tumor specific cohort to further evaluate antitumor activity of palbociclib in combination with topotecan and cyclophosphamide in children, adolescents, and young adults with recurrent or refractory neuroblastoma. Phase 2 to learn about the efficacy of palbociclib in combination with irinotecan and temozolomide when compared with irinotecan and temozolomide alone in the treatment of children, adolescents, and young adults with recurrent or refractory Ewing sarcoma (EWS).NCT03709680
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Principal InvestigatorAliceLee
Trial DescriptionStudy of Patients Receiving DUPIXENT® for Atopic Dermatitis (AD) - A long-term observational registry in patients with atopic dermatitis (AD) initiating treatment with DUPIXENT® (dupilumab)NCT03428646
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Principal InvestigatorHollyKanavy
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Trial DescriptionStudy of Sparsentan in Patients With Primary Focal Segmental Glomerulosclerosis (FSGS) - To determine the long-term nephroprotective potential of treatment with sparsentan as compared to an angiotensin receptor blocker in patients with primary and genetic focal segmental glomerulosclerosis (FSGS).NCT03493685
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Principal InvestigatorFrederickKaskel
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Trial DescriptionStudy of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) - The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.NCT02500381
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Principal InvestigatorVolkanGranit
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Trial DescriptionStudy to Assess PT010 in Adult and Adolescent Participants With Inadequately Controlled Asthma (LOGOS) - This is a variable length study to evaluate the efficacy and safety of budesonide/glycopyrronium/formoterol inhaler in adults and adolescents with severe asthma inadequately controlled with standard of care.NCT04609904
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Principal InvestigatorGoldaHudes
Trial DescriptionStudy to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031 - Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical TrialsNCT03573882
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Principal InvestigatorCaterinaMinniti
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Trial DescriptionStudy to Assess the Effectiveness and Safety of Lenacapavir for Human Immunodeficiency Virus (HIV) Pre-Exposure Prophylaxis - The goal of this clinical study is to test how well the study drug, lenacapavir (LEN), works in preventing the risk of HIV.NCT04925752
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Principal InvestigatorMichelleCollins-Ogle
Trial DescriptionStudy to Evaluate the Pharmacokinetics, Safety, and Antiviral Activity of the Elvitegravir/Cobicistat/Emtricitabine/Tenofovir Alafenamide (E/C/F/TAF) Single Tablet Regimen (STR) in HIV-1 Infected Antiretroviral Treatment-Naive Adolescents and Virolog - The primary objectives of Cohort 1 are to evaluate the steady state pharmacokinetics (PK) for elvitegravir (EVG) and tenofovir alafenamide (TAF) and confirm the dose of the elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide (E/C/F/TAF) STR (Part A) and to evaluate the safety and tolerability of E/C/F/TAF STR through Week 24 (Part B) in human immunodeficiency virus - 1 (HIV-1) infected, antiretroviral (ARV) treatment-naive adolescents. The primary objectives of Cohort 2 are to evaluate the PK of EVG and TAF in virologically suppressed HIV-1 infected children 6 to < 12 years of age weighing ≥ 25 kg administered E/C/F/TAF STR (Part A) and to evaluate the safety and tolerability of E/C/F/TAF STR through Week 24 in virologically suppressed HIV-1 infected children 6 to < 12 years of age weighing ≥ 25 kg (Part B). The primary objectives of Cohort 3 are to evaluate the PK of EVG and TAF and confirm the dose of the STR, and to evaluate the safety and tolerability of E/C/F/TAF low dose (LD) STR in virologically suppressed HIV-1 infected children ≥ 2 years of age and weighing ≥ 14 to < 25 kg.NCT01854775
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Principal InvestigatorDonnaFutterman
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Trial DescriptionStudy to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Extended-release Tablets in Pediatric Subjects Aged 5 to 17 Years With Active, Mild to Moderate Ulcerative Colitis - This is a randomized, double-blind, placebo-controlled, multicenter, study to evaluate the efficacy, safety, and pharmacokinetics (PK) of budesonide extended-release tablets for the induction of remission in pediatric subjects, with active, mild to moderate ulcerative colitis (UC). Subjects will be permitted to continue taking background oral or rectal 5-aminosalicylate (5-ASA) products.NCT04314375
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Principal InvestigatorGititTomer
Trial DescriptionStudy to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2- - This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged ≥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden. Randomization will be stratified by age groups (2 toNCT02435212
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Principal InvestigatorDeepaManwani
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Trial DescriptionSurveillance HeartCare® Outcomes Registry - This is an observational registry to assess the clinical utility of surveillance using HeartCare testing services, in association with clinical care of heart transplant recipients.NCT03695601
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Principal InvestigatorSnehalPatel
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Trial DescriptionSynCardia Freedom Driver System Study - The purposes of this study are to confirm that the Freedom Driver System is a suitable pneumatic driver for clinically stable TAH-t subjects, and that patients and lay caregivers can be trained to manage the Freedom Driver System safely outside the hospital.NCT00733447
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Principal InvestigatorDanielGoldstein
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Trial DescriptionTabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Participants With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease (EBV+ PTLD) After Failure of Rituximab or Rituximab and Chemotherapy - The purpose of this study is to determine the clinical benefit and characterize the safety profile of tabelecleucel for the treatment of Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) in the setting of (1) solid organ transplant (SOT) after failure of rituximab and rituximab plus chemotherapy or (2) allogeneic hematopoietic cell transplant (HCT) after failure of rituximab.NCT03394365
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Principal InvestigatorIoannisMantzaris
Trial DescriptionTacrolimus/Everolimus vs. Tacrolimus/MMF in Pediatric Heart Transplant Recipients Using the MATE Score - The TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will receive everolimus and low-dose tacrolimus and the other half will receive tacrolimus and mycophenolate mofetil. The trial will determine which treatment is better at reducing the cumulative risk of coronary artery vasculopathy, chronic kidney disease and biopsy proven-acute cellular rejection without an increase in graft loss due to all causes (e.g. infection, PTLD, antibody mediated rejection).NCT03386539
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Principal InvestigatorDaphneHsu
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Trial DescriptionTargeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial - This ComboMATCH patient screening trial is the gateway to a coordinated set of clinical trials to study cancer treatment directed by genetic testing. Patients with solid tumors that have spread to nearby tissue or lymph nodes (locally advanced) or have spread to other places in the body (advanced) and have progressed on at least one line of standard systemic therapy or have no standard treatment that has been shown to prolong overall survival may be candidates for these trials. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with some genetic changes or abnormalities (mutations) may benefit from treatment that targets that particular genetic mutation. ComboMATCH is designed to match patients to a treatment that may work to control their tumor and may help doctors plan better treatment for patients with locally advanced or advanced solid tumors.NCT05564377
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Principal InvestigatorBalazsHalmos
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Trial DescriptionTargeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial) - This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of standard systemic therapy and/or for which no standard treatment exists that has been shown to prolong survival. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic changes or abnormalities (mutations) may benefit more from treatment which targets their tumor's particular genetic mutation, and may help doctors plan better treatment for patients with solid tumors or non-Hodgkin lymphomas.NCT03155620
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Principal InvestigatorLisaGennarini
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Trial DescriptionTelehealth CBT for Adolescents and Young Adults With Childhood-onset Systemic Lupus Erythematosus - This study aims to investigate the feasibility and effectiveness of a remotely delivered psychological intervention for youth with cSLE. This intervention aims to teach participants skills in order to cope with fatigue, pain, and depressive symptoms--symptoms that commonly affect adolescents and young adults with lupus.NCT04335643
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Principal InvestigatorTamarRubinstein
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Trial DescriptionTelehealth Intervention for Autism Spectrum Disorder - Autism Spectrum Disorder (ASD) is one of the most frequently occurring childhood-onset neurodevelopmental disorders affecting 1 in every 54 children. Most children with ASD experience challenges participating in daily activities (e.g.: eating, sleeping, bathing, grooming, playing, etc.) and receive occupational therapy intervention to address these. The COVID-19 pandemic has restricted in-person therapy for many of these children and there is an urgent need for evidence-based, validated telehealth intervention. This project will adapt an evidence-based occupational therapy intervention, termed OT4ASD to a telehealth delivery model. The aims of the project are to: 1) adapt the existing intervention protocol to a telehealth delivery model, 2) train therapists and evaluate the therapist's ability to conduct OT4ASD, 3) determine if OT4ASD delivered via the telehealth is acceptable and feasible to parents and interventionists; and 4) whether children improve in the daily living skills. OT4ASD follows a systematic protocol and uses active, individually-tailored sensory motor activities that are specifically designed to address the child's needs. The investigators believe this will be the first telehealth manualized protocol to address the sensory motor symptoms of ASD and measure outcomes at the daily life activity and participation levels.NCT04832152
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Principal InvestigatorElizabethRidgway
Trial DescriptionTenofovir Alafenamide (TAF) in Children and Adolescents With Chronic Hepatitis B Virus Infection - The goals of this clinical study are to compare the effectiveness, safety and tolerability of study drug, tenofovir alafenamide (TAF), versus placebo in teens and children with CHB and to learn more about the dosing levels in children.NCT02932150
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Principal InvestigatorDebraPan
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Trial DescriptionThe B'N Fit POWER Initiative: A School-Based Wellness Initiative for Bronx Youth - The findings of this study will establish the feasibility and efficacy of B'N Fit POWER and given the wide presence of MSHP, MMCC and other afterschool programs, there is significant potential for dissemination of B'N Fit POWER to many other clinic-community partnership sites enabling the development of a larger grant that will test the effectiveness and dissemination process to several schools in the Bronx. The translation of a traditional clinical-based weight-loss intervention to adapting and implementing such an intervention in a real-world school setting is more relevant and sustainable for advancing a culture of health and promoting diabetes risk reduction in Bronx youth.NCT03565744
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Principal InvestigatorJessicaRieder
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Trial DescriptionThe iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study - This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling may result in identifying changes in genes of the tumor that indicate that a particular therapy may have activity. This is called an individualized cancer therapy (iCat) recommendation. The results of the tumor profiling and, if applicable, the iCat recommendation will be returned.NCT02520713
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Principal InvestigatorDanielWeiser
Trial DescriptionThe Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lymphoma Society and Children's Oncology Group Study - This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults.NCT04726241
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Principal InvestigatorLisaGennarini
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Trial DescriptionThe REPLACE Registry for Cholbam® (Cholic Acid) - This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using Cholbam for at least 30 days (existing users) and those who are first-time initiators of Cholbam.NCT03115086
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Principal InvestigatorNadiaOvchinsky
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Trial DescriptionThe Tetherâ„¢ - Vertebral Body Tethering System Post Approval Study - This study is an opportunity to provide continued reasonable assurance of the safety and probable benefit of The Tether HUD. The study will collect long term safety and efficacy information from patients who have had their idiopathic scoliosis treated via anterior vertebral body tethering (AVBT) with The Tether.NCT04505579
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Principal InvestigatorJaimeGomez
Trial DescriptionThoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma - This phase III trial compares the effect of open thoracic surgery (thoracotomy) to thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic surgery is a type of surgery done through a single larger incision (like a large cut) that goes between the ribs, opens up the chest, and removes the cancer. Thoracoscopy is a type of chest surgery where the doctor makes several small incisions and uses a small camera to help with removing the cancer. This trial is being done evaluate the two different surgery methods for patients with osteosarcoma that has spread to the lung to find out which is better.NCT05235165
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Principal InvestigatorLisaGennarini
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Trial DescriptionTipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial - This phase II pediatric MATCH trial studies how well tipifarnib works in treating patients with solid tumors that have recurred or spread to other places in the body (advanced), lymphoma, or histiocytic disorders, that have a genetic alteration in the gene HRAS. Tipifarnib may block the growth of cancer cells that have specific genetic changes in a gene called HRAS and may reduce tumor size.NCT04284774
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Principal InvestigatorLisaGennarini
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Trial DescriptionTransvenous Approach for the Treatment of Cerebral Arteriovenous Malformations - A new endovascular route for the treatment of brain AVMs may be possible in some cases: Trans-Venous Embolization (TVE). The technique uses microcatheters to navigate to the draining veins of AVM, to reach and then fill the AVM nidus retrogradely with liquid embolic agents until the lesion is occluded. This technique has the potential to improve on some of the problems with the arterial approach to AVM embolization, such as a low overall occlusion rate. However, by occluding the vein first, and filling the lesion with the embolic agent in a retrograde fashion, the method transgresses a widely held dogma in the surgical or endovascular treatment of AVMs: to preserve the draining vein until all afferent vessels have been occluded. Nevertheless, the initial case series have shown promising results, with high occlusion rates, and few technical complications. The method is increasingly used in an increasing number of centers, but there is currently no research protocol to guide the use of this promising but still experimental treatment in a prudent fashion. Care trials can be designed to offer such an experimental treatment, taking into account the best medical interests of patients, in the presence of rapidly evolving indications and techniques.NCT03691870
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Principal InvestigatorDavidAltschul
Trial DescriptionTreatment of Hemophilia A Patients With FVIII Inhibitors - This is a non-interventional, multicenter, observational, international study in male persons with haemophilia A who have developed inhibitors to any replacement coagulation factor VIII (FVIII) product. The purpose of the study is to capture different approaches in the management of persons with haemophilia A and FVIII inhibitors, document current immune tolerance induction approaches, and evaluate the efficacy and safety of immune tolerance induction, including the combination of FVIII and emicizumab. Patients will be assigned to 1 of 3 groups based on the treatments they receive, and may switch to another group if their treatment is changed. Participants will be followed after a maximum observational period of 5 years.NCT04023019
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Principal InvestigatorWilliamMitchell
Trial DescriptionTreatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents - Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in children. The cancer comes from a cell in the blood called a lymphocyte. Normal lymphocytes are produced in the bone marrow (along with other blood cells) and help fight infections. In ALL, the cancerous lymphocytes are called lymphoblasts. They do not help fight infection and crowd out the normal blood cells in the bone marrow so that the body cannot make enough normal blood cells. ALL is always fatal if it is not treated. With current treatments, most children and adolescents with this disease will be cured. The standard treatment for ALL involves about 2 years of chemotherapy. The drugs that are used, and the doses of the drugs, are similar but not identical for all children and adolescents with ALL. Some children and adolescents receive stronger treatment, especially during the first several months. A number of factors are used to decide how strong the treatment should be to give the best chance for cure. These factors are called "risk factors". This trial is studying the use of a new, updated set of risk factors to decide how strong the treatment will be. The study also will test a new way of dosing a chemotherapy drug called pegaspargase (which is part of the standard treatment for ALL) based on checking levels of the drug in the blood and adjusting the dose based on the levels.NCT03020030
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Principal InvestigatorLisaGennarini
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Trial DescriptionTreatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL) - This phase II trial studies if dinutuximab, GM-CSF, isotretinoin in combination with irinotecan, and temozolomide (chemo-immunotherapy) can be given safely to patients with high-risk neuroblastoma after Consolidation therapy (which usually consists of two autologous stem cell transplants and radiation) who have not experienced worsening or recurrence of their disease. Dinutuximab represents a kind of cancer therapy called immunotherapy. Unlike chemotherapy and radiation, dinutuximab targets the cancer cells without destroying nearby healthy cells. Sargramostim helps the body produce normal infection-fighting white blood cells. Isotretinoin helps the neuroblastoma cells become more mature. These 3 drugs (standard immunotherapy) are already given to patients with high-risk neuroblastoma after Consolidation because they have been proven to be beneficial in this setting. Chemotherapy drugs, such as irinotecan and temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. They may also affect how well immunotherapy works on neuroblastoma cells. Giving chemo-immunotherapy after intensive therapy may work better in treating patients with high-risk neuroblastoma compared to standard immunotherapy.NCT04385277
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Principal InvestigatorLisaGennarini
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Trial DescriptionTretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia - This phase III trial studies tretinoin and arsenic trioxide in treating patients with newly diagnosed acute promyelocytic leukemia. Standard treatment for acute promyelocytic leukemia involves high doses of a common class of chemotherapy drugs called anthracyclines, which are known to cause long-term side effects, especially to the heart. Tretinoin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Arsenic trioxide may stop the growth of cancer cells by either killing the cells, by stopping them from dividing, or by stopping them from spreading. Completely removing or reducing the amount of anthracycline chemotherapy and giving tretinoin together with arsenic trioxide may be an effective treatment for acute promyelocytic leukemia and may reduce some of the long-term side effects.NCT02339740
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Principal InvestigatorLisaGennarini
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Trial DescriptionTrial of Nivolumab Following Partially Human Leukocyte Antigen (HLA) Mismatched BMT in Children & Adults With Sarcoma - This research is being done to find out if an investigational drug, Nivolumab, can be safely administered after a "half-matched" (haplo) bone marrow transplant (BMT), and if the investigational drug will help to prevent or delay relapse or progression of sarcomas. In this study investigators will also be trying to learn more about how the investigational drug changes blood and/or tumors. Participants are eligible for this trial if they have recently undergone a "half-matched" (haplo) bone marrow transplant and have either relapsed or are at high risk to relapse.NCT03465592
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Principal InvestigatorDavidLoeb
Trial DescriptionTrial of Patterned Oral Somatosensory Entrainment for Shortening Time to Oral Feeding - The primary hypothesis is that the preterm infants (26 0/7 to 30 6/7 weeks gestational age) who undergo the NTrainer System® training will transition to full oral feeds faster than the control group (i.e. the study group will be superior to the control). The secondary hypothesis is that the infants in the NTrainer System® experimental group will have shorter lengths of stay.NCT01158391
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Principal InvestigatorSuhasNafday
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Trial DescriptionTrialNet Pathway to Prevention of T1D - Rationale: The accrual of data from the laboratory and from epidemiologic and prevention trials has improved the understanding of the etiology and pathogenesis of type 1 diabetes mellitus (T1DM). Genetic and immunologic factors play a key role in the development of T1DM, and characterization of the early metabolic abnormalities in T1DM is steadily increasing. However, information regarding the natural history of T1DM remains incomplete. The TrialNet Natural History Study of the Development of T1DM (Pathway to Prevention Study) has been designed to clarify this picture, and in so doing, will contribute to the development and implementation of studies aimed at prevention of and early treatment in T1DM. Purpose: TrialNet is an international network dedicated to the study, prevention, and early treatment of type 1 diabetes. TrialNet sites are located throughout the United States, Canada, Finland, United Kingdom, Italy, Germany, Sweden, Australia, and New Zealand. TrialNet is dedicated to testing new approaches to the prevention of and early intervention for type 1 diabetes. The goal of the TrialNet Natural History Study of the Development of Type 1 Diabetes is to enhance our understanding of the demographic, immunologic, and metabolic characteristics of individuals at risk for developing type 1 diabetes. The Natural History Study will screen relatives of people with type 1 diabetes to identify those at risk for developing the disease. Relatives of people with type 1 diabetes have about a 5% percent chance of being positive for the antibodies associated with diabetes. TrialNet will identify adults and children at risk for developing diabetes by testing for the presence of these antibodies in the blood. A positive antibody test is an early indication that damage to insulin-secreting cells may have begun. If this test is positive, additional testing will be offered to determine the likelihood that a person may develop diabetes. Individuals with antibodies will be offered the opportunity for further testing to determine their risk of developing diabetes over the next 5 years and to receive close monitoring for the development of diabetes.NCT00097292
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Principal InvestigatorLisaUnderland
Trial DescriptionTumor-Agnostic Precision Immuno-Oncology and Somatic Targeting Rational for You (TAPISTRY) Platform Study - TAPISTRY is a Phase II, global, multicenter, open-label, multi-cohort study designed to evaluate the safety and efficacy of targeted therapies or immunotherapy as single agents or in rational, specified combinations in participants with unresectable, locally advanced or metastatic solid tumors determined to harbor specific oncogenic genomic alterations or who are tumor mutational burden (TMB)-high as identified by a validated next-generation sequencing (NGS) assay. Participants with solid tumors will be treated with a drug or drug regimen tailored to their NGS assay results at screening. Participants will be assigned to the appropriate cohort based on their genetic alteration(s). Treatment will be assigned on the basis of relevant oncogenotype, will have cohort-specific inclusion/exclusion criteria, and, unless otherwise specified, will continue until disease progression, loss of clinical benefit, unacceptable toxicity, participant or physician decision to discontinue, or death, whichever occurs first.NCT04589845
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Principal InvestigatorHaiyingCheng
Trial DescriptionUse of a Probiotic Supplement to Prevent Asthma in Infants - The goal of the study is to understand the mechanisms of how antigen presentation affects the developing immune system and subsequently affects susceptibility to, or protects against, asthma development. This randomized controlled study will test the effectiveness of daily supplementation of Lactobacillus GG for the first 6 months of life on the early immunological development of asthma.NCT00113659
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Principal InvestigatorMichaelCabana
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Trial DescriptionVeliparib, Radiation Therapy, and Temozolomide in Treating Patients With Newly Diagnosed Malignant Glioma Without H3 K27M or BRAFV600 Mutations - This phase II trial studies how well veliparib, radiation therapy, and temozolomide work in treating patients with newly diagnosed malignant glioma without H3 K27M or BRAFV600 mutations. Poly adenosine diphosphate (ADP) ribose polymerases (PARPs) are proteins that help repair DNA mutations. PARP inhibitors, such as veliparib, can keep PARP from working, so tumor cells can't repair themselves, and they may stop growing. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving veliparib, radiation therapy, and temozolomide may work better in treating patients with newly diagnosed malignant glioma without H3 K27M or BRAFV600 mutations compared to radiation therapy and temozolomide alone.NCT03581292
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Principal InvestigatorLisaGennarini
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Trial DescriptionViral Infection and Respiratory Illness Universal Study[VIRUS]: COVID-19 Registry - Researchers are creating a real time COVID-19 registry of current ICU/hospital care patterns to allow evaluations of safety and observational effectiveness of COVID-19 practices and to determine the variations in practice across hospitals.NCT04323787
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Principal InvestigatorJen-TingChen
Trial DescriptionVirtual Reality Goggle Utilization for Venipuncture Distraction - This is a randomized non-blinded trial evaluating the effect of virtual reality goggles on perceived pain and anxiety scores during venipuncture on hospitalized children ages 5-21. We will randomized patients to either standard of care (including Lidocaine 2.5%/Prilocaine 2.5% cream as a topical anesthetic 60-240 minutes prior to venipuncture) or standard of care plus virtual reality goggles.NCT04479735
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Principal InvestigatorCourtneyMcnamara
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