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Research

Clinical Trials

CHAM researchers are involved in literally hundreds of clinical trials and research studies to evaluate the effectiveness and safety of innovative treatments and diagnostics. Search and refine for clinical trials and research studies at CHAM by keyword or disease/condition.

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Asthma

A 52-week, Randomised, Double-blind, Placebo-controlled, Parallel-group, Multi-centre Study of the Efficacy and Safety of GSK3511294 Adjunctive Therapy in Adult and Adolescent Participants With Severe Uncontrolled Asthma With an Eosinophilic Phenotype

Participants will be randomized in a 2:1 ratio to receive either GSK3511294 (Depemokimab) or placebo as an adjunct therapy.

Principal Investigator: Golda Hudes
Inflammatory Bowel Diseases, Crohn's Disease, Ulcerative Colitis, Indeterminate Colitis

A 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease

Principal Investigator: Gitit Tomer
Acute Lymphoblastic Leukemia

A Comprehensive Approach to Improve Medication Adherence in Pediatric ALL

Principal Investigator: Lisa Gennarini
Duchenne Muscular Dystrophy

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Principal Investigator: Volkan Granit
Duchenne Muscular Dystrophy

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Principal Investigator: Volkan Granit
Biliary Atresia

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy

Principal Investigator: Debra Pan
Nonalcoholic Fatty Liver, Nonalcoholic Steatohepatitis

A Longitudinal Observational Study of Patients With Nonalcoholic Steatohepatitis (NASH) and Related Conditions Across the Entire Spectrum of Nonalcoholic Fatty Liver Disease (NAFLD)

Principal Investigator: Preeti Viswanathan
Juvenile Idiopathic Arthritis

A LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)

Principal Investigator: Dawn Wahezi
Juvenile Idiopathic Arthritis

A LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)

Principal Investigator: Dawn Wahezi
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications

Principal Investigator: David Loeb
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications

Principal Investigator: David Loeb
Infusion Reactions

A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients

Principal Investigator: David Loeb
Infusion Reactions

A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients

Principal Investigator: David Loeb
Pulmonary Arterial Hypertension

A Multicenter, Open-label, Randomized, Study With Single-arm Extension Period to Assess the Pharmacokinetics, Safety and Efficacy of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension

Principal Investigator: Nicole Sutton
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndrome

A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic Syndrome (MDS)

Principal Investigator: Alice Lee
Crohn's Disease, Ulcerative Colitis, Indeterminate Colitis, Inflammatory Bowel Diseases

A Multicenter, Prospective, Long-term, Observational Registry of Pediatric Patients With Inflammatory Bowel Disease

Principal Investigator: Gitit Tomer
Systemic Lupus Erythematosus

A Multi-center, Randomized, Placebo-Controlled Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of Belimumab, a Human Monoclonal Anti-BLyS Antibody, Plus Standard Therapy in Pediatric Patients With Systemic Lupus Erythematosus

Principal Investigator: Dawn Wahezi
Systemic Lupus Erythematosus

A Multi-center, Randomized, Placebo-Controlled Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of Belimumab, a Human Monoclonal Anti-BLyS Antibody, Plus Standard Therapy in Pediatric Patients With Systemic Lupus Erythematosus

Principal Investigator: Dawn Wahezi
Asthma

A Multi-centre, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety of GSK3511294 (Depemokimab) in Adult and Adolescent Participants With Severe Asthma With an Eosinophilic Phenotype From Studies 206713 or 213744

Principal Investigator: Golda Hudes
Pediatric Sarcoma, Refractory, Pediatric Sarcoma, Relapsed

A 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

Single arm, unblinded, phase I/II study

Principal Investigator: Alice Lee
Ovarian Cancer, Endometrial Cancer, Germ Cell Tumor, Testicular Germ Cell Tumor, Ovarian Germ Cell Tumor

A Phase 1, First-in-Human, Dose Escalation and Expansion Study to Evaluate the Safety and Tolerability of XmAb®541 in Advanced Solid Tumors

Dose escalation and expansion study

Principal Investigator: Eric Feldman
Nosocomial Pneumonia

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

Principal Investigator: Michelle Collins-Ogle
Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia, Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma

A Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-X19 in Pediatric and Adolescent Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4)

Principal Investigator: Ellen Fraint
Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia, Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma

A Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-X19 in Pediatric and Adolescent Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4)

Principal Investigator: Ellen Fraint
Anaplastic Astrocytoma, Anaplastic Astrocytoma, Not Otherwise Specified, Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Glioblastoma, Glioblastoma, Not Otherwise Specified, Malignant Glioma

A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)

Principal Investigator: Alice Lee
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Mixed Lineage Acute Leukemia, Mixed Phenotype Acute Leukemia, Acute Leukemia of Ambiguous Lineage

A Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients With Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation

Phase 1 will employ an accelerated titration design. The dose escalation will follow a modified Fibonacci sequence.

Principal Investigator: Ioannis Mantzaris
Recurrent B Acute Lymphoblastic Leukemia, Recurrent B Lymphoblastic Lymphoma, Refractory B Acute Lymphoblastic Leukemia, Refractory B Lymphoblastic Lymphoma

A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518) in Children and Young Adults With Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)

Principal Investigator: Alice Lee
Medulloblastoma

A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients

Principal Investigator: Lisa Gennarini
Leukemia

A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway-Mutant Acute Lymphoblastic Leukemia

Principal Investigator: Lisa Gennarini
Leukemia

A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway-Mutant Acute Lymphoblastic Leukemia

Principal Investigator: Lisa Gennarini
Anaplastic Astrocytoma, Glioblastoma, Malignant Glioma

A Phase 2 Study of Veliparib (ABT-888) and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients With Newly Diagnosed High-Grade Glioma (HGG) Without H3 K27M or BRAFV600 Mutations

Principal Investigator: Lisa Gennarini
Central Nervous System Nongerminomatous Germ Cell Tumor, Choriocarcinoma, Embryonal Carcinoma, Immature Teratoma, Malignant Teratoma, Mixed Germ Cell Tumor, Pineal Region Germ Cell Tumor, Pineal Region Immature Teratoma, Pineal Region Yolk Sac Tumor, Suprasellar Germ Cell Tumor

A Phase 2 Trial of Chemotherapy Followed by Response-Based Whole Ventricular &Amp; Spinal Canal Irradiation (WVSCI) for Patients With Localized Non-Germinomatous Central Nervous System Germ Cell Tumor

Principal Investigator: Lisa Gennarini
Sickle Cell Disease (SCD)

A Phase 2,Multicenter,Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab,With or Without Hydroxyurea/Hydroxycarbamide,in Sequential,Descending Age Groups of Pediatric Sickle Cell Disease Patients With Vaso-Occlusive Crisis

Principal Investigator: Deepa Manwani
Proteinuric Kidney Disease

A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease

Principal Investigator: Michael Ross
Heart Failure, Left Ventricular Systolic Dysfunction

A Phase 2/3 Randomized, Placebo-Controlled, Double-blind, Clinical Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Systemic Left Ventricular Systolic Dysfunction (VALOR)

Principal Investigator: Daphne Hsu
Acquired Immune Deficiency Syndrome (AIDS), HIV Infections

A Phase 2/3, Open-Label Study of 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 Virologically Suppressed Children

Principal Investigator: Donna Futterman
Acquired Immune Deficiency Syndrome (AIDS), HIV Infections

A Phase 2/3, Open-Label Study of 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 Virologically Suppressed Children

Principal Investigator: Donna Futterman
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)

A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity

All participants will receive mirdametinib (PD-0325901) at a dose of 2 mg/m^2 twice daily (maximum dose of 4 mg twice daily), calculated based on body surface area. Dose will be administered in a 3-week on, 1-week off schedule.

Principal Investigator: Nagma Dalvi
Ulcerative Colitis (UC)

A Phase 3 Multicenter, Long-Term Extension Study to Evaluate the Safety and Efficacy of Upadacitinib (ABT-494) in Subjects With Ulcerative Colitis

Principal Investigator: Thomas Ullman
Arthritis, Juvenile

A Phase 3 Multicenter, Open-label Study to Evaluate the Efficacy, Pharmacokinetics, Safety, and Immunogenicity of Subcutaneously Administered Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic Arthritis (PSUMMIT-Jr)

Principal Investigator: Dawn Wahezi
Arthritis, Juvenile

A Phase 3 Multicenter, Open-label Study to Evaluate the Efficacy, Pharmacokinetics, Safety, and Immunogenicity of Subcutaneously Administered Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic Arthritis (PSUMMIT-Jr)

Principal Investigator: Dawn Wahezi
Metastatic Malignant Neoplasm in the Lung, Metastatic Osteosarcoma, Osteosarcoma

A Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma

Principal Investigator: Alice Lee
Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma

A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)

Principal Investigator: Lisa Gennarini
Acute Myeloid Leukemia

A Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations

Principal Investigator: Alice Lee
Central Nervous System Carcinoma

A Phase 3 Randomized, Placebo-Controlled Trial Evaluating Memantine for Neurocognitive Protection in Children Undergoing Cranial Radiotherapy as Part of Treatment for Primary Central Nervous System Tumors

Principal Investigator: Alice Lee
Atopic Dermatitis

A Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Upadacitinib in Adolescent and Adult Subjects With Moderate to Severe Atopic Dermatitis

Principal Investigator: Holly Kanavy
Sickle Cell Disease

A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the BB305 Lentiviral Vector in Subjects With Sickle Cell Disease

Principal Investigator: Deepa Manwani
Ganglioneuroblastoma, Neuroblastoma

A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)

Principal Investigator: Alice Lee
Pre-Exposure Prophylaxis of HIV Infection

A Phase 3, Double-Blind, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Subcutaneous Twice Yearly Long-Acting Lenacapavir for HIV Pre-Exposure Prophylaxis in Cisgender Men, Transgender Women, Transgender Men, and Gender Nonbinary People ≥ 16 Years of Age Who Have Sex With Male Partners and Are at Risk for HIV Infection

Principal Investigator: Michelle Collins-Ogle
Areata Alopecia, Alopecia, Hypotrichosis, Hair Diseases, Skin Diseases, Pathological Conditions, Anatomical

A Phase 3, Double-Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics (PK) of Baricitinib in Children From 6 Years to Less Than 18 Years of Age With Alopecia Areata

Principal Investigator: Holly Kanavy
Contraception

A Phase 3, Multicenter, Open-Label, Single Arm Study of MR-100A-01 in Women of Childbearing Potential to Evaluate Contraceptive Efficacy and Safety

Principal Investigator: Jessica Atrio
Contraception

A Phase 3, Multicenter, Open-Label, Single Arm Study of MR-100A-01 in Women of Childbearing Potential to Evaluate Contraceptive Efficacy and Safety

Principal Investigator: Jessica Atrio
Immune Thrombocytopenia

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

Principal Investigator: Irina Murakhovskaya
Contraception

A Phase 3, Open-label, Multi-center, Single Arm Study to Assess Contraceptive Efficacy and Safety of the Etonogestrel (MK-8415) Implant During Extended Use From 3 Years After Insertion in Females 35 Years of Age or Younger

Principal Investigator: Jessica Atrio
Pediatric Growth Hormone Deficiency

A Phase 3, Open-label, Randomized, Multicenter, 12 Months, Efficacy and Safety Study of Weekly MOD-4023 Compared to Daily Genotropin - Therapy in Pre-pubertal Children With Growth Hormone Deficiency

Principal Investigator: Ping Zhou
Pediatric Growth Hormone Deficiency

A Phase 3, Open-label, Randomized, Multicenter, 12 Months, Efficacy and Safety Study of Weekly MOD-4023 Compared to Daily Genotropin - Therapy in Pre-pubertal Children With Growth Hormone Deficiency

Principal Investigator: Ping Zhou
Atopic Dermatitis

A Phase 3, Randomized, 52-week, Placebo-controlled, Double-blind Study With Rerandomization to Assess the Efficacy, Safety, and Tolerability of Rocatinlimab (AMG 451) in Adolescent Subjects With Moderate-to-severe Atopic Dermatitis (AD) (ROCKET-ASTRO)

Principal Investigator: Holly Kanavy
Non-Cystic Fibrosis Bronchiectasis

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib Administered Once Daily for 52 Weeks in Subjects With Non-Cystic Fibrosis Bronchiectasis - The ASPEN Study

Principal Investigator: Divya Reddy
Migraine

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Rimegepant in Migraine Prevention in Children and Adolescents ≥ 6 to <18 Years of Age

Principal Investigator: Jelena Pavlovic
Chronic Kidney Disease; Iron Deficiency Anemia

A Phase 3, Randomized, Open-Label, Multicenter 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)

Principal Investigator: Anna Zolotnitskaya
Psoriasis

A Phase 3b, Multi Center, Open-label, Long-term Extension Study of Apremilast (CC-10004) in Pediatric Subjects From 6 Through 17 Years of Age With Moderate to Severe Plaque Psoriasis

Principal Investigator: Julia Gittler
Atopic Dermatitis

A Phase 3b/4 Randomized, Open-label, Efficacy Assessor Blinded Study, Comparing the Safety and Assessor Blinded Efficacy of Upadacitinib to Dupilumab in Subjects With Moderate to Severe Atopic Dermatitis (Level-Up)

Principal Investigator: Holly Kanavy
Atopic Dermatitis

A Phase 3b/4 Randomized, Open-label, Efficacy Assessor Blinded Study, Comparing the Safety and Assessor Blinded Efficacy of Upadacitinib to Dupilumab in Subjects With Moderate to Severe Atopic Dermatitis (Level-Up)

Principal Investigator: Holly Kanavy
Osteosarcoma, Osteosarcoma in Children, Osteosarcoma Recurrent, Sarcoma

A Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination With Azacitidine in Patients With Recurrent, Resectable Osteosarcoma

Phase Ib lead-in with extension to Phase II

Principal Investigator: Daniel Weiser
Autism Spectrum Disorder (ASD)

A Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of RO7017773 in Participants Aged 15-45 Years With Autism Spectrum Disorder (ASD)

Principal Investigator: Eric Hollander
Refractory and/or Relapsed Metastatic Solid Tumors

A Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors

Principal Investigator: David Loeb
Melanoma of Unknown Primary, Recurrent Melanoma, Stage IIIB Cutaneous Melanoma AJCC v7, Stage IIIC Cutaneous Melanoma AJCC v7, Stage IV Cutaneous Melanoma AJCC v6 and v7

A Phase III Randomized Study of Adjuvant Ipilimumab Anti-CTLA4 Therapy Versus High-Dose Interferon a-2b for Resected High-Risk Melanoma

Principal Investigator: Joseph Sparano
Anaplastic Medulloblastoma, Medulloblastoma, Supratentorial Embryonal Tumor, Not Otherwise Specified, Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor

A Phase III Randomized Trial for the Treatment of Newly Diagnosed Supratentorial PNET and High Risk Medulloblastoma in Children < 36 Months Old With Intensive Induction Chemotherapy With Methotrexate Followed by Consolidation With Stem Cell Rescue vs. the Same Therapy Without Methotrexate

Principal Investigator: Jonathan Gill
Acute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA

A Phase III Study for Patients With Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid

Principal Investigator: Lisa Gennarini
Acute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA

A Phase III Study for Patients With Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid

Principal Investigator: Lisa Gennarini
Ganglioneuroblastoma, Nodular, Neuroblastoma

A Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma

Principal Investigator: Lisa Gennarini
Dermatitis, Atopic

A Prospective Observational Study of Patients Receiving DUPIXENT® for Atopic Dermatitis

Principal Investigator: Holly Kanavy
Dermatitis, Atopic

A Prospective Observational Study of Patients Receiving DUPIXENT® for Atopic Dermatitis

Principal Investigator: Holly Kanavy
Embryonal Rhabdomyosarcoma, Fusion-Negative Alveolar Rhabdomyosarcoma, Spindle Cell/Sclerosing Rhabdomyosarcoma

A Prospective Phase 3 Study of Patients With Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma

Principal Investigator: Alice Lee
Spinal Muscular Atrophy (SMA)

A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Principal Investigator: LESLIE DELFINER
Spinal Muscular Atrophy (SMA)

A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Principal Investigator: LESLIE DELFINER
Short Bowel Syndrome

A Prospective, Multi-center Registry for Patients With Short Bowel Syndrome

Principal Investigator: John Thompson
Short Bowel Syndrome

A Prospective, Multi-center Registry for Patients With Short Bowel Syndrome

Principal Investigator: John Thompson
Bile Acid Synthesis Disorders

A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid)

Principal Investigator: Nadia Ovchinsky
Chondral Defect, Osteochondritis Dissecans (OCD), Articular Cartilage Defect, Articular Cartilage Disorder of Knee

A Prospective, Open-label, Randomized, Concurrent Active-controlled, Longitudinal, Multicenter, Phase 3 Clinical Study of the Safety and Efficacy of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee

Principal Investigator: Eric Fornari
HIV/AIDS

A Randomized Controlled Trial of Women Involved in Supporting Health (WISH), a Peer-Led Intervention to Improve Postpartum Retention in HIV Care

Participants will be assigned in equal proportions to either the WISH Adherence arm or to the WISH Parenting arm

Principal Investigator: Rodney Wright
HIV/AIDS

A Randomized Controlled Trial of Women Involved in Supporting Health (WISH), a Peer-Led Intervention to Improve Postpartum Retention in HIV Care

Participants will be assigned in equal proportions to either the WISH Adherence arm or to the WISH Parenting arm

Principal Investigator: Rodney Wright
Lugano Classification Limited Stage Hodgkin Lymphoma AJCC v8

A Randomized Phase 3 Interim Response Adapted Trial Comparing Standard Therapy With Immuno-oncology Therapy for Children and Adults With Newly Diagnosed Stage I and II Classic Hodgkin Lymphoma

Principal Investigator: Alice Lee
Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Rhabdomyosarcoma, Sclerosing Rhabdomyosarcoma, Spindle Cell Rhabdomyosarcoma

A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)

Principal Investigator: Alice Lee
Primary Mediastinal Large B-Cell Lymphoma

A Randomized Phase 3 Trial of Nivolumab (NSC# 748726) in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma

Principal Investigator: Alice Lee
Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Metastatic Embryonal Rhabdomyosarcoma, Metastatic Rhabdomyosarcoma, Solid Alveolar Rhabdomyosarcoma, Spindle Cell Rhabdomyosarcoma, Spindle Cell/Sclerosing Rhabdomyosarcoma

A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy With Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) Plus VINO-CPO Maintenance in Patients With High Risk Rhabdomyosarcoma (HR-RMS)

Principal Investigator: Alice Lee
Germ Cell Tumor, Teratoma, Choriocarcinoma, Germinoma, Mixed Germ Cell Tumor, Yolk Sac Tumor, Childhood Teratoma, Malignant Germ Cell Neoplasm, Extragonadal Seminoma, Non-seminomatous Germ Cell Tumor, Seminoma

A Randomized Phase III Trial Comparing Conventional-Dose Chemotherapy Using Paclitaxel, Ifosfamide, and Cisplatin (TIP) With High-Dose Chemotherapy Using Mobilizing Paclitaxel Plus Ifosfamide Followed by High-Dose Carboplatin and Etoposide (TI-CE) as First Salvage Treatment in Relapsed or Refractory Germ Cell Tumors

Principal Investigator: Benjamin Gartrell
B Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, B Acute Lymphoblastic Leukemia, BCR-ABL1-Like, Lymphoblastic Lymphoma, Mixed Phenotype Acute Leukemia, T Acute Lymphoblastic Leukemia

A Randomized Trial of Levocarnitine Prophylaxis to Prevent Asparaginase-Associated Hepatotoxicity in Adolescents and Young Adults Receiving Acute Lymphoblastic Leukemia Therapy

Principal Investigator: Alice Lee
Bowel Preparation

A Randomized, Assessor-Blind, Parallel-Groups, Multicenter Trial Assessing the Safety and Efficacy, Including Pharmacokinetic Assessments, of CLENPIQ in Children Aged 2 Years to Less Than 9 Years

Principal Investigator: Inna Novak
Necrotizing Enterocolitis

A Randomized, Double Blind, Parallel-group, Placebo Controlled Study to Evaluate the Efficacy and Safety of IBP-9414 in Premature Infants 500-1500g Birth Weight in the Prevention of Necrotizing Enterocolitis - The Connection Study

Principal Investigator: Shantanu Rastogi
Chronic Hepatitis B

A Randomized, Double-Blind Evaluation of the Pharmacokinetics, Safety, and Antiviral Efficacy of Tenofovir Alafenamide (TAF) in Children and Adolescent Subjects With Chronic Hepatitis B Virus Infection

Principal Investigator: Debra Pan
Seizures, Lennox Gastaut Syndrome

A Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Carisbamate (YKP509) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults, With Optional Open-Label Extension

This double-blind, randomized, placebo-controlled study will evaluate the efficacy of carisbamate 200 mg BID or the pediatric equivalent dose and 300 mg BID or the pediatric equivalent dose for the treatment of seizures associated with Lennox Gastaut syndrome in subjects 4 to 55 years of age.

Principal Investigator: Alexis Boro
Ulcerative Colitis

A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Extended-release Tablets n Pediatric Subjects Aged 5 to 17 Years With Active, Mild to Moderate Ulcerative Colitis

Principal Investigator: Gitit Tomer
Focal Segmental Glomerulosclerosis

A Randomized, Multicenter, Double-blind, Parallel, Active-control Study of the Effects of Sparsentan, a Dual Endothelin Receptor and Angiotensin Receptor Blocker, on Renal Outcomes in Patients With Primary FSGS

Principal Investigator: Frederick Kaskel
Acute Myeloid Leukemia (AML), Cancer

A Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in Combination With Azacitidine After Allogeneic Stem Cell Transplantation in Subjects With Acute Myeloid Leukemia (AML) (VIALE-T)

Principal Investigator: Ioannis Mantzaris
Systemic Lupus Erythematosus

A Remotely Delivered CBT Intervention for Youth With cSLE: A Multi-site Patient-engaged Investigation

Participants will either be randomly assigned to the intervention or to a wait-list period.

Principal Investigator: Tamar Rubinstein
Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis, Unresectable Desmoid Fibromatosis

A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors

Principal Investigator: Lisa Gennarini
Untreated Childhood Medulloblastoma

A Study Evaluating Limited Target Volume Boost Irradiation and Reduced Dose Craniospinal Radiotherapy (18.00 Gy) and Chemotherapy in Children With Newly Diagnosed Standard Risk Medulloblastoma: A Phase III Double Randomized Trial

Principal Investigator: Peter Cole
Breast Cancer

A Trial of Endocrine Response in Women With Invasive Lobular Breast Cancer

Principal Investigator: Sheldon Feldman
Breast Cancer

A Trial of Endocrine Response in Women With Invasive Lobular Breast Cancer

Principal Investigator: Sheldon Feldman
Aortic Stenosis

ACURATE IDE: Transcatheter Replacement of Stenotic Aortic Valve Through Implantation of ACURATE in Subjects InDicatEd for TAVR

Principal Investigator: Mohamed Azeem Latib
Sickle Cell Disease

An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Principal Investigator: Caterina Minniti
Sickle Cell Disease

An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Principal Investigator: Caterina Minniti
Juvenile Idiopathic Arthritis

An Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA)

Principal Investigator: Dawn Wahezi
Sickle Cell Disease

An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)

Principal Investigator: Henny Billett
Sickle Cell Disease

An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)

Principal Investigator: Henny Billett
Autism

An Open-Label Extension Study of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin

Principal Investigator: Eric Hollander
Autism

An Open-Label Extension Study of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin

Principal Investigator: Eric Hollander
Progressive Familial Intrahepatic Cholestasis (PFIC)

An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

Principal Investigator: Nadia Ovchinsky
Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia

An Open-label, First-in-human, Dose-escalation/Expansion Study of SAR443579 Administered as Single Agent by Intravenous Infusion in Adult and Pediatric Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Principal Investigator: Ioannis Mantzaris
Kidney Diseases, Kidney Failure, Kidney Disease, Chronic

APOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO)

Principal Investigator: Enver Akalin
Heart Transplant Infection, Kidney Transplant Infection, Liver Transplant Infection, CMV

Assessment of CMV-specific T Cell Responses by Flow Cytometry With Intracellular Cytokine Staining to Predict CMV Infection Risk in Pediatric Solid Organ Transplant Recipients

Principal Investigator: Betsy Herold
Acute Myeloid Leukemia, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IV Breast Cancer AJCC v8, Clinical Stage III Cutaneous Melanoma AJCC v8, Clinical Stage IV Cutaneous Melanoma AJCC v8, Clinical Stage IV Esophageal Adenocarcinoma AJCC v8, Clinical Stage IV Esophageal Squamous Cell Carcinoma AJCC v8, Clinical Stage IV Gastric Cancer AJCC v8, Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8, Lung Non-Small Cell Carcinoma, Lung Small Cell Carcinoma, Malignant Solid Neoplasm, Metastatic Prostate Carcinoma, Multiple Myeloma, Stage III Lung Cancer AJCC v8, Stage III Ovarian Cancer AJCC v8, Stage IV Colorectal Cancer AJCC v8, Stage IV Lung Cancer AJCC v8, Stage IV Ovarian Cancer AJCC v8, Stage IV Prostate Cancer AJCC v8, Stage IVB Prostate Cancer AJCC v8

Cancer Moonshot Biobank Research Protocol

Principal Investigator: Balazs Halmos
Acute Myeloid Leukemia, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IV Breast Cancer AJCC v8, Clinical Stage III Cutaneous Melanoma AJCC v8, Clinical Stage IV Cutaneous Melanoma AJCC v8, Clinical Stage IV Esophageal Adenocarcinoma AJCC v8, Clinical Stage IV Esophageal Squamous Cell Carcinoma AJCC v8, Clinical Stage IV Gastric Cancer AJCC v8, Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8, Lung Non-Small Cell Carcinoma, Lung Small Cell Carcinoma, Malignant Solid Neoplasm, Metastatic Prostate Carcinoma, Multiple Myeloma, Stage III Lung Cancer AJCC v8, Stage III Ovarian Cancer AJCC v8, Stage IV Colorectal Cancer AJCC v8, Stage IV Lung Cancer AJCC v8, Stage IV Ovarian Cancer AJCC v8, Stage IV Prostate Cancer AJCC v8, Stage IVB Prostate Cancer AJCC v8

Cancer Moonshot Biobank Research Protocol

Principal Investigator: Balazs Halmos
Prader-Willi Syndrome

Cannabidivarin (CBDV) vs. Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)

Principal Investigator: Eric Hollander
Autism Spectrum Disorder

Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)

Phase 2, 12-week double-blind, randomized, placebo-controlled trial

Principal Investigator: Eric Hollander
B Acute Lymphoblastic Leukemia Associated With Down Syndrome, Down Syndrome, Myeloid Leukemia Associated With Down Syndrome

Chronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)

Principal Investigator: Alice Lee
Chronic Kidney Disease

Chronic Kidney Disease in Children Prospective Cohort Study (CKiD)

Principal Investigator: Frederick Kaskel
Bronchiolitis

Comparison of Conventional Non-Invasive Ventilation and Neurally-Adjusted Ventilatory Assistance (NAVA) Non-Invasive Ventilation for the Treatment of Bronchiolitis

Non-blinded, randomized clinical trial

Principal Investigator: Jacqueline Weingarten-Arams
Sickle Cell Disease, COVID-19

COVID-19 Vaccine Response in People Living With Sickle Cell Disease

Principal Investigator: Deepa Manwani
Acute Lymphoblastic Leukemia

Documentation and Delivery of Guideline-Consistent Treatment in Adolescent and Young Adult (AYA) Acute Lymphoblastic Leukemia (ALL)

Principal Investigator: David Loeb
Lupus Nephritis

Efficacy & Safety of Pharmacokinetically-Driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis- A Double-Blind Placebo Controlled Clinical Trial

Eligible patients enrolled in the study will be randomized (1:1) at baseline to the 53-week double-blind, active comparator 2-part study to receive either MMFPK or MMFBSA. Subjects who are partial renal responders (PRR) to MMFBSA at week 26, will cross over to the MMFPK arm. Complete renal responders (CRR) at week 26 in MMFBSA arm will continue to be treated with MMFBSA. Subjects with at least a PRR (or even CRR) in the MMFPK arm at week 26 will remain in the MMFPK arm and continue to receive MMF dosage targeting MPA-AUC0-12 > 60-70 mg*h/l. Subjects whose LN fails to respond to therapy by week 26 will be discontinued from the study interventions to receive LN treatment as per their local physician's decision. Subjects who experience a single episode of a LN flare during Part 1 of the study or fulfill other criteria for discontinuation from the study intervention, will also receive LN treatment as per their local physician's decision.

Principal Investigator: Dawn Wahezi
Anaplastic Medulloblastoma, Medulloblastoma

Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

Principal Investigator: Lisa Gennarini
Bronchiolitis Acute Viral

Eliminating Monitor Overuse (EMO) Hybrid Effectiveness-Deimplementation Trial

Principal Investigator: Alyssa Silver
Bronchiolitis Acute Viral

Eliminating Monitor Overuse (EMO) Hybrid Effectiveness-Deimplementation Trial

Principal Investigator: Alyssa Silver
Malignant Neoplasm

EROS: Engendering Reproductive Health Within Oncologic Survivorship

Principal Investigator: Della Makower
Asthma in Children

Evaluation of the Asthma Management Program to Promote Activity for Students in Schools (Asthma-PASS)

Principal Investigator: Marina Reznik
Rhabdomyosarcoma

Evolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma

Principal Investigator: Alice Lee
DiGeorge Syndrome, 22q11.2 Deletion Syndrome

Genetic Modifiers of 22q11.2 Deletion Syndrome

Principal Investigator: Bernice Morrow
DiGeorge Syndrome, 22q11.2 Deletion Syndrome

Genetic Modifiers of 22q11.2 Deletion Syndrome

Principal Investigator: Bernice Morrow
Congenital Heart Disease

Implementing Models for Mechanical Circulatory Support Presurgical Assessment in Congenital Heart Disease Treatment

Principal Investigator: Omar Saeed
Anatomic Stage 0 Breast Cancer AJCC v8, Anatomic Stage I Breast Cancer AJCC v8, Anatomic Stage IA Breast Cancer AJCC v8, Anatomic Stage IB Breast Cancer AJCC v8, Anatomic Stage II Breast Cancer AJCC v8, Anatomic Stage IIA Breast Cancer AJCC v8, Anatomic Stage IIB Breast Cancer AJCC v8, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IIIA Breast Cancer AJCC v8, Anatomic Stage IIIB Breast Cancer AJCC v8, Anatomic Stage IIIC Breast Cancer AJCC v8, Prognostic Stage 0 Breast Cancer AJCC v8, Prognostic Stage I Breast Cancer AJCC v8, Prognostic Stage IA Breast Cancer AJCC v8, Prognostic Stage IB Breast Cancer AJCC v8, Prognostic Stage II Breast Cancer AJCC v8, Prognostic Stage IIA Breast Cancer AJCC v8, Prognostic Stage IIB Breast Cancer AJCC v8, Prognostic Stage III Breast Cancer AJCC v8, Prognostic Stage IIIA Breast Cancer AJCC v8, Prognostic Stage IIIB Breast Cancer AJCC v8, Prognostic Stage IIIC Breast Cancer AJCC v8

Improving Patient-Centered Communication in Breast Cancer: A RCT of a Shared Decision Engagement System (SHARES)

Principal Investigator: Sheldon Feldman
Anatomic Stage 0 Breast Cancer AJCC v8, Anatomic Stage I Breast Cancer AJCC v8, Anatomic Stage IA Breast Cancer AJCC v8, Anatomic Stage IB Breast Cancer AJCC v8, Anatomic Stage II Breast Cancer AJCC v8, Anatomic Stage IIA Breast Cancer AJCC v8, Anatomic Stage IIB Breast Cancer AJCC v8, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IIIA Breast Cancer AJCC v8, Anatomic Stage IIIB Breast Cancer AJCC v8, Anatomic Stage IIIC Breast Cancer AJCC v8, Prognostic Stage 0 Breast Cancer AJCC v8, Prognostic Stage I Breast Cancer AJCC v8, Prognostic Stage IA Breast Cancer AJCC v8, Prognostic Stage IB Breast Cancer AJCC v8, Prognostic Stage II Breast Cancer AJCC v8, Prognostic Stage IIA Breast Cancer AJCC v8, Prognostic Stage IIB Breast Cancer AJCC v8, Prognostic Stage III Breast Cancer AJCC v8, Prognostic Stage IIIA Breast Cancer AJCC v8, Prognostic Stage IIIB Breast Cancer AJCC v8, Prognostic Stage IIIC Breast Cancer AJCC v8

Improving Patient-Centered Communication in Breast Cancer: A RCT of a Shared Decision Engagement System (SHARES)

Principal Investigator: Sheldon Feldman
Aortic Valve Disease, Aortic Valve Insufficiency, Aortic Regurgitation

J-Valve TF Early Feasibility Study

Principal Investigator: Mohamed Azeem Latib
Neoplasms

Long-Term Follow-Up (LTFU) of Participants Treated With GSK Adoptive Cell Therapies

Principal Investigator: David Loeb
Neoplasms

Master Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered NY-ESO-1-Specific (c259) T Cells, Alone or in Combination With Other Agents, in HLA-A2+ Participants With NY-ESO-1 and/or LAGE-1a Positive Solid Tumors (IGNYTE-ESO)

Principal Investigator: David Loeb
Neoplasms

Master Protocol to Assess the Safety and Antitumor Activity of Genetically Engineered NY-ESO-1-Specific (c259) T Cells, Alone or in Combination With Other Agents, in HLA-A2+ Participants With NY-ESO-1 and/or LAGE-1a Positive Solid Tumors (IGNYTE-ESO)

Principal Investigator: David Loeb
Recurrent, Refractory, or High Risk Leukemias, Matched Targeted Therapy

Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome

Principal Investigator: Lisa Gennarini
Osteosarcoma, Ewing Sarcoma

Metformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse

Group 1 Localized osteosarcoma that have presented a response ≤ 60% to the pre-operatory chemotherapy. Is expected the enrolment of 37 patients as total population, in 44 months. The patients will be followed for 3 years, started from the date of enrolment. Metformin will be administrated for 3 years maximum, unless there is a progression disease or if toxicity is verified. Group 2 Osteosarcoma and Ewing sarcoma patients with complete remission after the first relapse. Is expected the enrolment of 30 patients as total population, in 24 months. Metformin will be administrated at the Group 2 for 1 year. If there is no toxicity verified or relapse, the patient should continue the treatment for other 2 year (in a total 3 years as maximum).

Osteosarcoma, Ewing Sarcoma

Metformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse

Group 1 Localized osteosarcoma that have presented a response ≤ 60% to the pre-operatory chemotherapy. Is expected the enrolment of 37 patients as total population, in 44 months. The patients will be followed for 3 years, started from the date of enrolment. Metformin will be administrated for 3 years maximum, unless there is a progression disease or if toxicity is verified. Group 2 Osteosarcoma and Ewing sarcoma patients with complete remission after the first relapse. Is expected the enrolment of 30 patients as total population, in 24 months. Metformin will be administrated at the Group 2 for 1 year. If there is no toxicity verified or relapse, the patient should continue the treatment for other 2 year (in a total 3 years as maximum).

Sickle Cell Disease

Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease

Principal Investigator: Michelle Lee
Hemophilia A

MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study

Principal Investigator: William Mitchell
Hemophilia A

MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study

Principal Investigator: William Mitchell
Advanced Malignant Solid Neoplasm, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IV Breast Cancer AJCC v8, Locally Advanced Malignant Solid Neoplasm, Malignant Female Reproductive System Neoplasm, Metastatic HER2-Negative Breast Carcinoma, Metastatic Malignant Solid Neoplasm, Recurrent Endometrial Carcinoma, Recurrent Fallopian Tube Carcinoma, Recurrent Malignant Female Reproductive System Neoplasm, Recurrent Malignant Solid Neoplasm, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma, Unresectable HER2-Negative Breast Carcinoma, Unresectable Malignant Solid Neoplasm

Molecular Analysis for Combination Therapy Choice (ComboMATCH)

Principal Investigator: Balazs Halmos
Head and Neck Neoplasms

Molecular and Genomic Profiling of Head and Neck Tumors to Explore Biomarkers Associated With Prognosis and Response to Therapy

Principal Investigator: Richard Smith
Head and Neck Neoplasms

Molecular and Genomic Profiling of Head and Neck Tumors to Explore Biomarkers Associated With Prognosis and Response to Therapy

Principal Investigator: Richard Smith
Childhood Obesity, Epigenetics

Molecular Basis of Early Childhood Obesity Programming by Intrauterine Growth Restriction

Principal Investigator: Mamta Fuloria
Inflammatory Bowel Disease

Multi-Center African-American Inflammatory Bowel Disease Study (MAAIS)

Principal Investigator: Ruby Greywoode
Pediatric Solid Tumor

Multicenter Cohort Study To Evaluate Outcomes After Receipt of Targeted Therapy Matched to an Individualized Cancer Therapy (iCat) Recommendations in Children and Young Adults With Solid Tumors: The iCat2, GAIN Consortium Study

Principal Investigator: Daniel Weiser
Pediatric Solid Tumor

Multicenter Cohort Study To Evaluate Outcomes After Receipt of Targeted Therapy Matched to an Individualized Cancer Therapy (iCat) Recommendations in Children and Young Adults With Solid Tumors: The iCat2, GAIN Consortium Study

Principal Investigator: Daniel Weiser
Primary Immunodeficiency Diseases (PID)

Multicenter, Prospective, Open-label, Randomized, Crossover Study to Evaluate Pharmacokinetics (PK), Safety, and Tolerability of TAK-881 in Primary Immunodeficiency Diseases (PIDD)

This study consists of crossover randomized epoch (participants aged >=16 years) and single arm epoch (participants aged 2 to <16 years).

Principal Investigator: Arye Rubinstein
Neoplasms, Arteriovenous Malformation of Central Nervous System, Trigeminal Neuralgia

Multi-Institution Registry of SRS/SBRT Procedures

Principal Investigator: Madhur Garg
Neoplasms, Arteriovenous Malformation of Central Nervous System, Trigeminal Neuralgia

Multi-Institution Registry of SRS/SBRT Procedures

Principal Investigator: Madhur Garg
Hypoplastic Left Heart Syndrome (HLHS)

National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children With Complex Congenital Heart Disease

Principal Investigator: Nicole Sutton
Hypoplastic Left Heart Syndrome (HLHS)

National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children With Complex Congenital Heart Disease

Principal Investigator: Nicole Sutton
Mycobacterium Infections

Natural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections

Principal Investigator: Steven Porcelli
COVID-19 Infection, Hematopoietic and Lymphatic System Neoplasm, Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm

NCI COVID-19 in Cancer Patients Study (NCCAPS): A Longitudinal Natural History Study

Principal Investigator: Lisa Gennarini
Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations

Principal Investigator: Lisa Gennarini
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Osteosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Refractory Childhood Malignant Germ Cell Tumor, Refractory Childhood Osteosarcoma, Refractory Childhood Rhabdomyosarcoma, Refractory Childhood Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Wilms Tumor

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Erdafitinib in Patients With Tumors Harboring FGFR1/2/3/4 Alterations

Principal Investigator: Lisa Gennarini
Advanced Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Kidney Wilms Tumor, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-101 (Larotrectinib) in Patients With Tumors Harboring Actionable NTRK Fusions

Principal Investigator: Lisa Gennarini
Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Histiocytic and Dendritic Cell Neoplasm, Recurrent Langerhans Cell Histiocytosis, Recurrent Lymphoma, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Histiocytic and Dendritic Cell Neoplasm, Refractory Langerhans Cell Histiocytosis, Refractory Lymphoma, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-292 in Patients With Tumors Harboring RET Gene Alterations

Principal Investigator: Lisa Gennarini
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Wilms Tumor

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Ensartinib in Patients With Tumors Harboring ALK or ROS1 Genomic Alterations

Principal Investigator: Lisa Gennarini
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Malignant Glioma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Rhabdoid Tumor, Stage III Osteosarcoma AJCC v7, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Osteosarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Stage IVA Osteosarcoma AJCC v7, Stage IVB Osteosarcoma AJCC v7, Wilms Tumor

NCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice)- Phase 2 Subprotocol of LY3023414 in Patients With Solid Tumors

Principal Investigator: Lisa Gennarini
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma, Recurrent Ectomesenchymoma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Kidney Wilms Tumor, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Medulloblastoma, Recurrent Melanoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdoid Tumor of the Kidney, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Carcinoma, Recurrent WHO Grade 2 Glioma, Refractory Adrenal Gland Pheochromocytoma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Medulloblastoma, Refractory Melanoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdoid Tumor of the Kidney, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Carcinoma, Refractory WHO Grade 2 Glioma

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Tipifarnib in Patients With Tumors Harboring HRAS Genomic Alterations

Principal Investigator: Lisa Gennarini
Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma

Neuroblastoma Biology Studies

Principal Investigator: Lisa Gennarini
Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma

Neuroblastoma Biology Studies

Principal Investigator: Lisa Gennarini
Neonatal Hypoglycemia, Neurodevelopmental Disorders

Neurodevelopmental Outcome of Normoglycemic Versus Hypoglycemic Neonates at Risk for Hypoglycemia

Principal Investigator: Mariam Susan Latuga
Neonatal Hypoglycemia, Neurodevelopmental Disorders

Neurodevelopmental Outcome of Normoglycemic Versus Hypoglycemic Neonates at Risk for Hypoglycemia

Principal Investigator: Mariam Susan Latuga
Childhood Malignant Neoplasm

Neuropsychological, Social, Emotional, and Behavioral Outcomes in Children With Cancer

Principal Investigator: Lisa Gennarini
Rheumatic Joint Disease

Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

Principal Investigator: Dawn Wahezi
Rheumatic Joint Disease

Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

Principal Investigator: Dawn Wahezi
H3 K27M, Glioma

ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study

Principal Investigator: Adilia Hormigo
Symptomatic Aortic Stenosis

Optimize PRO Transcatheter Aortic Valve Replacement Post Market Study

Principal Investigator: Mohamed Azeem Latib
Pain, Procedural

Optimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure?

randomized non blinded study

Principal Investigator: Courtney Mcnamara
Acute Kidney Injury, Chronic Kidney Disease

Outcomes of Neonatal Acute Kidney Injury and Prematurity: An Observational Study and Einstein-Montefiore Neonatal Acute Kidney Injury Clinical Registry and Biorepository

Principal Investigator: Mamta Fuloria
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Juvenile Myelomonocytic Leukemia, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome Post Cytotoxic Therapy, Myeloid Leukemia Associated With Down Syndrome

Pediatric Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias

Principal Investigator: Alice Lee
Childhood Hepatocellular Carcinoma, Childhood Malignant Liver Neoplasm, Fibrolamellar Carcinoma, Hepatoblastoma, Hepatocellular Malignant Neoplasm, Not Otherwise Specified

Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

Principal Investigator: Alice Lee
Ewing Sarcoma, Solid Tumors, Rhabdoid Tumor, Rhabdomyosarcoma, Neuroblastoma, Medulloblastoma, Diffuse Intrinsic Pontine Glioma

PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS

Phase 1 portion: The palbociclib plus irinotecan and temozolomide combination part of the study will comprise of a dose escalation cohort (following a rolling 6 design), a dose expansion cohort. The palbociclib plus topotecan and cyclophosphamide will comprise of a dose determination cohort(following a modified rolling 6 design), a dose expansion cohort, and Neuroblastoma tumor -specific cohort. Phase 2 open-label, randomized portion of the study will randomize patients in a 2:1 ratio to receive either palbociclib in combination with irinotecan and temozolomide (Arm A) or irinotecan and temozolomide chemotherapy alone (Arm B). Randomization will be stratified using block randomization by type and time of current disease recurrence (primary refractory or 1st recurrence < 2 years versus 1st recurrence ≥ 2 years or 2nd or greater recurrence).

Principal Investigator: Alice Lee
Sickle Cell Disease, Pain

Phase 1-2 Trial of Gamunex (Intravenous Gammaglobulin) for Sickle Cell Acute Pain

Principal Investigator: Deepa Manwani
Sickle Cell Disease, Pain

Phase 1-2 Trial of Gamunex (Intravenous Gammaglobulin) for Sickle Cell Acute Pain

Principal Investigator: Deepa Manwani
Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis

Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis

Principal Investigator: Alice Lee
Prader-Willi Syndrome, Hyperphagia

Phase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

Principal Investigator: Eric Hollander
Central Nervous System Nongerminomatous Germ Cell Tumor, Childhood Central Nervous System Germinoma

Phase 2 Trial of Response-Based Radiation Therapy for Patients With Localized Central Nervous System Germ Cell Tumors (CNS GCT)

Principal Investigator: Lisa Gennarini
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic

Phase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders

Principal Investigator: Michelle Lee
Osteosarcoma, Ewing Sarcoma, Rhabdomyosarcoma, Soft Tissue Sarcoma

Phase II Study of Nab-Paclitaxel in Combination With Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults

Principal Investigator: Daniel Weiser
Pediatric Heart Transplantation, Immunosuppression, Chronic Kidney Diseases, Cardiac Allograft Vasculopathy, Heart Transplant Failure and Rejection, Post-transplant Lymphoproliferative Disorder, Heart Transplant Infection

Phase III Multicenter Open-label Randomized Clinical Trial Comparing Everolimus and Low Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil at 6 mo Post-Transplant to Prevent Long-term Complications After Pediatric Heart Transplantation

Multicenter open-label randomized clinical trial with randomization within 4 strata, defined by donor-specific antibody status and center annual transplant volume. There are 2 parallel groups of equal sizes for randomization: everolimus/low-dose tacrolimus and tacrolimus/mycophenolate mofetil.

Principal Investigator: Daphne Hsu
Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Recurrent Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma

Phase III Randomized Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue

Principal Investigator: Jonathan Gill
Asthma Childhood

Promoting Asthma Guidelines and Management Through Technology-Based Intervention and Care Coordination

enhanced Usual Care and Intervention sites

Principal Investigator: Marina Reznik
COVID-19 Infection, Hematopoietic and Lymphatic System Neoplasm, Malignant Solid Neoplasm

Prospective Cohort Study to Evaluate Immunologic Response Following COVID-19 Vaccination in Children, Adolescents and Young Adults With Cancer

Principal Investigator: Alice Lee
Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation, Solid Tumors, Blood Cancers, CAR-T, Gene Therapy, Non-malignant Disease

Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

Principal Investigator: Michelle Lee
Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation, Solid Tumors, Blood Cancers, CAR-T, Gene Therapy, Non-malignant Disease

Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

Principal Investigator: Michelle Lee
Cmv Congenital, CMV, Congenital Cmv, SNHL, Sensorineural Hearing Loss

Randomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial

Principal Investigator: Michel Nassar
Metastatic Ewing Sarcoma, Metastatic Malignant Neoplasm in the Bone, Metastatic Malignant Neoplasm in the Bone Marrow, Metastatic Malignant Neoplasm in the Lung, Metastatic Peripheral Primitive Neuroectodermal Tumor of Bone, Peripheral Primitive Neuroectodermal Tumor of Soft Tissues

Randomized Phase 3 Trial Evaluating the Addition of the IGF-1R Monoclonal Antibody Ganitumab (AMG 479, NSC# 750008) to Multiagent Chemotherapy for Patients With Newly Diagnosed Metastatic Ewing Sarcoma

Principal Investigator: Alice Lee
Acute Ischemic Stroke, Vessel Occlusion, Stroke, Ischemic

REal-World Analyses of Stroke - Thrombus Occlusion REtrieval

Principal Investigator: Neil Haranhalli
Asthma

Registry of Asthma Patients Initiating DUPIXENT® (RAPID)

Principal Investigator: Golda Hudes
Adult 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

Renal 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.

Principal Investigator: Lisa Gennarini
Hodgkin Disease

Risk-based, Response-adapted, Phase II Open-label Trial of Nivolumab + Brentuximab Vedotin (N + Bv) for Children, Adolescents, and Young Adults With Relapsed/Refractory (R/R) CD30 + Classic Hodgkin Lymphoma (cHL) After Failure of First-line Therapy, Followed by Brentuximab + Bendamustine (Bv + B) for Participants With a Suboptimal Response (CheckMate 744: CHECKpoint Pathway and Nivolumab Clinical Trial Evaluation)

Principal Investigator: Lisa Gennarini
Acute Myeloid Leukemia, Down Syndrome, Myelodysplastic Syndrome, Myeloid Leukemia Associated With Down Syndrome, Myeloproliferative Neoplasm

Risk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

Principal Investigator: Lisa Gennarini
Aortic Stenosis, Severe

Safety and Effectiveness of Balloon-Expandable Bioprosthetic SAPIEN X4 Transcatheter Heart Valve

Principal Investigator: Mohamed Azeem Latib
Sickle Cell Disease

Sickle Cell Disease (SCD) Biochip': Towards a Simple and Reliable Way to Monitor Sickle Cell Disease

Principal Investigator: Deepa Manwani
Bone Cyst

Simple Bone Cysts in Kids (SBoCK)

Principal Investigator: Jacob Schulz
Bone Cyst

Simple Bone Cysts in Kids (SBoCK)

Principal Investigator: Jacob Schulz
Sarcoma, Solid Tumor, Adult, Solid Tumor, Childhood

Single-arm, Open-label, Phase 1b/2 Trial of Nivolumab Therapy Following Partially HLA Mismatched (Haploidentical) Bone Marrow Transplant in Children and Young Adults With High Risk, Recurrent or Refractory Sarcomas

Adults: 240 mg IV (in a vein) over 30 minutes every 2 weeks OR 480 mg IV over 30 minutes every 4 weeks. Children and Adolescents weighing 40 kg or more: 240 mg IV over 30 minutes every 2 weeks OR 480 mg IV over 30 minutes every 4 weeks. Children and Adolescents weighing less than 40 kg: 3 mg/kg IV over 30 minutes every 2 weeks. A maximum of 24 cycles will be given on study. Participants may continue to receive Nivolumab unless they develop serious side effects or the tumor worsens. There were two parts to this study. The first part, Part A, was for patients who have relapsed or have progressive disease after their BMT. Part A is now closed. The second part, Part B, is for patients who have not yet relapsed or progressed after BMT.

Principal Investigator: David Loeb
Chronic Phase Chronic Myeloid Leukemia, BCR-ABL1 Positive

Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP)

Principal Investigator: Alice Lee
Heart Transplant Rejection

Surveillance HeartCare® Outcomes Registry

Principal Investigator: Snehal Patel
Autism Spectrum Disorder

Testing the Feasibility of a Teletherapy Plus Parent Coaching Intervention for Children With Autism

The purpose of this project is to adapt an existing, evidence-based occupational therapy intervention for children with ASD to a telehealth delivery model. The main objectives of the project are to 1) adapt the manual so that it operationalizes best-practices of occupational therapy telehealth50,51, 2) test its feasibility, and 3) evaluate preliminary outcomes. Design: In the formative research aspect of this project will use survey methodology and a modified Delphi process to adapt our current intervention to a telehealth delivery model (Aim 1). In Aim 2 we will conduct a feasibility trial (Figure 2) to assess therapists' fidelity, participant recruitment and retention, acceptability of the protocol by parents and therapists, and clinically meaningful differences.

Principal Investigator: Elizabeth Ridgway
Bone Marrow Transplantation

The Collection of Research Samples and/or Data for Repository From Related or Unrelated Hematopoietic Stem Cell Transplantation Recipients for the National Marrow Donor Program

Principal Investigator: Ira Braunschweig
Bone Marrow Transplantation

The Collection of Research Samples and/or Data for Repository From Related or Unrelated Hematopoietic Stem Cell Transplantation Recipients for the National Marrow Donor Program

Principal Investigator: Ira Braunschweig
Carcinoma In Situ, Central Nervous System Neoplasm, Childhood Immature Teratoma, Childhood Langerhans Cell Histiocytosis, Childhood Mature Teratoma, Congenital Mesoblastic Nephroma, Desmoid Fibromatosis, Ganglioneuroma, Lymphoproliferative Disorder, Malignant Solid Neoplasm, Melanocytic Neoplasm, Myeloproliferative Neoplasm, Neuroendocrine Neoplasm, Stromal Neoplasm

The Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study

Principal Investigator: Alice Lee
Carcinoma In Situ, Central Nervous System Neoplasm, Childhood Immature Teratoma, Childhood Langerhans Cell Histiocytosis, Childhood Mature Teratoma, Congenital Mesoblastic Nephroma, Desmoid Fibromatosis, Ganglioneuroma, Lymphoproliferative Disorder, Malignant Solid Neoplasm, Melanocytic Neoplasm, Myeloproliferative Neoplasm, Neuroendocrine Neoplasm, Stromal Neoplasm

The Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study

Principal Investigator: Alice Lee
Arteriovenous Malformations, Cerebral, Unruptured Brain Arteriovenous Malformation, Ruptured Brain Arteriovenous Malformation

Transvenous Approach for the Treatment of Cerebral Arteriovenous Malformations (TATAM): A Randomized Controlled Trial and Registry

Principal Investigator: David Altschul
Adult Kidney Wilms Tumor, Beckwith-Wiedemann Syndrome, Childhood Kidney Wilms Tumor, Diffuse Hyperplastic Perilobar Nephroblastomatosis, Rhabdoid Tumor of the Kidney, Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor, Stage V Kidney Wilms Tumor

Treatment for Patients With Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor

Principal Investigator: Lisa Gennarini
Acute Lymphoblastic Leukemia, Pediatric

Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents

Principal Investigator: Lisa Gennarini
Anaplastic Kidney Wilms Tumor, Recurrent Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor

Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)

Principal Investigator: Alice Lee
Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma, Ann Arbor Stage I B Lymphoblastic Lymphoma, Ann Arbor Stage II B Lymphoblastic Lymphoma, Childhood B Acute Lymphoblastic Leukemia, Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, Childhood B Lymphoblastic Lymphoma, Down Syndrome, Hypodiploid B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Positive

Treatment of Patients With Newly Diagnosed Standard Risk B-Lymphoblastic Leukemia (B-ALL) or Localized B-Lineage Lymphoblastic Lymphoma (B-LLy)

Principal Investigator: Alice Lee
Asthma

Trial of Infant Probiotic Supplementation to Prevent Asthma

Principal Investigator: Michael Cabana
Heart Transplant Rejection

Trifecta-Heart cfDNA-MMDX Study: Comparing the DD-cfDNA Test to MMDx Microarray Test and Central HLA Antibody Test

Principal Investigator: Snehal Patel
Solid Tumors

Tumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial

Principal Investigator: Haiying Cheng
Hematopoietic Cell Transplantation Recipient, Leukemia, Solid Tumor

Umbrella Long-Term Follow-Up Protocol

Principal Investigator: Alice Lee
Neurofibromatosis Type 1, Plexiform Neurofibromas

US Selumetinib Registry

Principal Investigator: Nagma Dalvi
Coronavirus

Viral Infection and Respiratory Illness Universal Study[VIRUS]: COVID-19 Registry and Validation of C2D2 (Critical Care Data Dictionary)

Principal Investigator: Jen-Ting Chen