Trial DescriptionA 52-week, Randomised, Double-blind, Double-dummy, Parallel Group, Multi-centre, Non-inferiority Study Assessing Exacerbation Rate, Additional Measures of Asthma Control and Safety in Adult and Adolescent Severe Asthmatic Participants With an Eosinophilic Phenotype Treated With GSK3511294 Compared With Mepolizumab or Benralizumab - This is randomized, double-blind, parallel group, multi-center and non-inferiority study.NCT04718389
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 52-week, Randomised, Double-blind, Double-dummy, Parallel Group, Multi-centre, Non-inferiority Study Assessing Exacerbation Rate, Additional Measures of Asthma Control and Safety in Adult and Adolescent Severe Asthmatic Participants With an Eosinophilic Phenotype Treated With GSK3511294 Compared With Mepolizumab or Benralizumab - This is randomized, double-blind, parallel group, multi-center and non-inferiority study.NCT04718389
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 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.NCT04718103
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 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 NCT02500381
Disease/ConditionDuchenne Muscular Dystrophy
Principal InvestigatorVolkan Granit
Trial DescriptionA 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 NCT04336722
Disease/ConditionBiliary Atresia
Principal InvestigatorDebra Pan
Trial DescriptionA Longitudinal Observational Study of Patients Undergoing Therapy for Immune-Mediated Inflammatory Skin Conditions NCT03661866
Disease/ConditionAtopic Dermatitis
Alopecia Areata
Hidradenitis Suppurativa
Vitiligo
Psoriasis
Chronic Spontaneous Urticaria
Principal InvestigatorHolly Kanavy
Trial DescriptionA Longitudinal Observational Study of Patients With Nonalcoholic Steatohepatitis (NASH) and Related Conditions Across the Entire Spectrum of Nonalcoholic Fatty Liver Disease (NAFLD) NCT02815891
Disease/ConditionNonalcoholic Fatty Liver
Nonalcoholic Steatohepatitis
Principal InvestigatorPreeti Viswanathan
Trial DescriptionA Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution NCT05044819
Disease/ConditionLennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Principal InvestigatorPuja Patel
Trial DescriptionA Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution NCT05044819
Disease/ConditionLennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Principal InvestigatorPuja Patel
Trial DescriptionA LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA) NCT01500551
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
Trial DescriptionA LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA) NCT01500551
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
Trial DescriptionA 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 NCT01351545
Disease/ConditionHematologic 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
Principal InvestigatorDavid Loeb
Trial DescriptionA Multicenter Pilot Study of Reduced Intensity Conditioning and Allogeneic Stem Cell Transplantation Followed by Targeted Immunotherapy in Children, Adolescents and Young Adults With Poor Risk CD30+ Hodgkin Lymphoma (HL) NCT02098512
Disease/ConditionHodgkin Lymphoma
Principal InvestigatorKris Mahadeo
Trial DescriptionA 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 NCT01656603
Disease/ConditionInfusion Reactions
Principal InvestigatorDavid Loeb
Trial DescriptionA 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 NCT02932410
Disease/ConditionPulmonary Arterial Hypertension
Principal InvestigatorNicole Sutton
Trial DescriptionA 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) NCT05457556
Disease/ConditionAcute Lymphoblastic Leukemia
Acute Myeloid Leukemia
Mixed Phenotype Acute Leukemia
Myelodysplastic Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionA Multicenter, Prospective, Long-term, Observational Registry of Pediatric Patients With Inflammatory Bowel Disease NCT00606346
Disease/ConditionCrohn's Disease
Ulcerative Colitis
Indeterminate Colitis
Inflammatory Bowel Diseases
Principal InvestigatorGitit Tomer
Trial DescriptionA 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 NCT05243680
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
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 - Single arm, unblinded, phase I/II studyNCT05634369
Disease/ConditionPediatric Sarcoma, Refractory
Pediatric Sarcoma, Relapsed
Principal InvestigatorAlice Lee
Trial DescriptionA 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.NCT04065399
Disease/ConditionAcute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Mixed Lineage Acute Leukemia
Mixed Phenotype Acute Leukemia
Acute Leukemia of Ambiguous Lineage
Principal InvestigatorIoannis Mantzaris
Trial DescriptionA Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma NCT03794349
Disease/ConditionHigh Risk Neuroblastoma
Recurrent Neuroblastoma
Refractory Neuroblastoma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG) NCT03919071
Disease/ConditionAnaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Anaplastic Ganglioglioma
Anaplastic Pleomorphic Xanthoastrocytoma
Glioblastoma
Malignant Glioma
WHO Grade 3 Glioma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG) NCT03919071
Disease/ConditionAnaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Anaplastic Ganglioglioma
Anaplastic Pleomorphic Xanthoastrocytoma
Glioblastoma
Malignant Glioma
WHO Grade 3 Glioma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518) in Children and Young Adults With Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL) NCT02981628
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Recurrent B Lymphoblastic Lymphoma
Refractory B Acute Lymphoblastic Leukemia
Refractory B Lymphoblastic Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients NCT02724579
Disease/ConditionMedulloblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA 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 NCT02723994
Disease/ConditionLeukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA 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 NCT03581292
Disease/ConditionAnaplastic Astrocytoma
Glioblastoma
Malignant Glioma
Principal InvestigatorLisa Gennarini
Trial DescriptionA 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 NCT05312879
Disease/ConditionProteinuric Kidney Disease
Principal InvestigatorMichael Ross
Trial DescriptionA 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) NCT05714085
Disease/ConditionHeart Failure
Left Ventricular Systolic Dysfunction
Principal InvestigatorDaphne Hsu
Trial DescriptionA 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 NCT01854775
Disease/ConditionAcquired Immune Deficiency Syndrome (AIDS)
HIV Infections
Principal InvestigatorDonna Futterman
Trial DescriptionA 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.NCT03962543
Disease/ConditionPlexiform Neurofibroma
Neurofibromatosis Type 1 (NF1)
Principal InvestigatorNagma Dalvi
Trial DescriptionA 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.NCT03962543
Disease/ConditionPlexiform Neurofibroma
Neurofibromatosis Type 1 (NF1)
Principal InvestigatorNagma Dalvi
Trial DescriptionA Phase 3 Multicenter, Long-Term Extension Study to Evaluate the Safety and Efficacy of Upadacitinib (ABT-494) in Subjects With Ulcerative Colitis NCT03006068
Disease/ConditionUlcerative Colitis (UC)
Principal InvestigatorThomas Ullman
Trial DescriptionA 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) NCT05083182
Disease/ConditionArthritis, Juvenile
Principal InvestigatorDawn Wahezi
Trial DescriptionA 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) NCT04166409
Disease/ConditionLow Grade Astrocytoma
Low Grade Glioma
Metastatic Low Grade Astrocytoma
Metastatic Low Grade Glioma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) NCT03871257
Disease/ConditionLow Grade Glioma
Neurofibromatosis Type 1
Visual Pathway Glioma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) NCT03871257
Disease/ConditionLow Grade Glioma
Neurofibromatosis Type 1
Visual Pathway Glioma
Principal InvestigatorLisa Gennarini
Trial DescriptionA 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 NCT04293562
Disease/ConditionAcute Myeloid Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Randomized Trial of Inotuzumab Ozogamicin (NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy NCT03959085
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Central Nervous System Leukemia
Mixed Phenotype Acute Leukemia
Testicular Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionA 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 NCT04939597
Disease/ConditionCentral Nervous System Carcinoma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Upadacitinib in Adolescent and Adult Subjects With Moderate to Severe Atopic Dermatitis NCT03569293
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
Trial DescriptionA 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 NCT04293185
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Trial DescriptionA 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 NCT04293185
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Trial DescriptionA 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) NCT03126916
Disease/ConditionGanglioneuroblastoma
Neuroblastoma
Principal InvestigatorAlice Lee
Trial DescriptionA 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) NCT03126916
Disease/ConditionGanglioneuroblastoma
Neuroblastoma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients With Germ Cell Tumors NCT03067181
Disease/ConditionChildhood Extracranial Germ Cell Tumor
Extragonadal Embryonal Carcinoma
Germ Cell Tumor
Malignant Germ Cell Tumor
Malignant Ovarian Teratoma
Stage I Ovarian Choriocarcinoma
Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage I Ovarian Teratoma AJCC v6 and v7
Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage I Testicular Choriocarcinoma AJCC v6 and v7
Stage I Testicular Embryonal Carcinoma AJCC v6 and v7
Stage I Testicular Seminoma AJCC v6 and v7
Stage I Testicular Yolk Sac Tumor AJCC v6 and v7
Stage II Ovarian Choriocarcinoma
Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage II Testicular Choriocarcinoma AJCC v6 and v7
Stage II Testicular Embryonal Carcinoma AJCC v6 and v7
Stage II Testicular Yolk Sac Tumor AJCC v6 and v7
Stage III Ovarian Choriocarcinoma
Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage III Testicular Choriocarcinoma AJCC v6 and v7
Stage III Testicular Embryonal Carcinoma AJCC v6 and v7
Stage III Testicular Yolk Sac Tumor AJCC v6 and v7
Stage IV Ovarian Choriocarcinoma
Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7
Testicular Mixed Choriocarcinoma and Embryonal Carcinoma
Testicular Mixed Choriocarcinoma and Teratoma
Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients With Germ Cell Tumors NCT03067181
Disease/ConditionChildhood Extracranial Germ Cell Tumor
Extragonadal Embryonal Carcinoma
Germ Cell Tumor
Malignant Germ Cell Tumor
Malignant Ovarian Teratoma
Stage I Ovarian Choriocarcinoma
Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage I Ovarian Teratoma AJCC v6 and v7
Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage I Testicular Choriocarcinoma AJCC v6 and v7
Stage I Testicular Embryonal Carcinoma AJCC v6 and v7
Stage I Testicular Seminoma AJCC v6 and v7
Stage I Testicular Yolk Sac Tumor AJCC v6 and v7
Stage II Ovarian Choriocarcinoma
Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage II Testicular Choriocarcinoma AJCC v6 and v7
Stage II Testicular Embryonal Carcinoma AJCC v6 and v7
Stage II Testicular Yolk Sac Tumor AJCC v6 and v7
Stage III Ovarian Choriocarcinoma
Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage III Testicular Choriocarcinoma AJCC v6 and v7
Stage III Testicular Embryonal Carcinoma AJCC v6 and v7
Stage III Testicular Yolk Sac Tumor AJCC v6 and v7
Stage IV Ovarian Choriocarcinoma
Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7
Testicular Mixed Choriocarcinoma and Embryonal Carcinoma
Testicular Mixed Choriocarcinoma and Teratoma
Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Trial Investigating Blinatumomab (NSC# 765986) in Combination With Chemotherapy in Patients With Newly Diagnosed Standard Risk or Down Syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients With Localized B-Lymphoblastic Lymphoma (B-LLy) NCT03914625
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Down Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Trial Investigating Blinatumomab (NSC# 765986) in Combination With Chemotherapy in Patients With Newly Diagnosed Standard Risk or Down Syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients With Localized B-Lymphoblastic Lymphoma (B-LLy) NCT03914625
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Down Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionA 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 NCT04925752
Disease/ConditionPre-Exposure Prophylaxis of HIV Infection
Principal InvestigatorMichelle Collins-Ogle
Trial DescriptionA 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 NCT05723198
Disease/ConditionAreata Alopecia
Alopecia
Hypotrichosis
Hair Diseases
Skin Diseases
Pathological Conditions, Anatomical
Principal InvestigatorHolly Kanavy
Trial DescriptionA Phase 3, Multicenter, Open-Label, Single Arm Study of MR-100A-01 in Women of Childbearing Potential to Evaluate Contraceptive Efficacy and Safety NCT05139121
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
Trial DescriptionA 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) NCT04562766
Disease/ConditionImmune Thrombocytopenia
Principal InvestigatorIrina Murakhovskaya
Trial DescriptionA 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 NCT04626596
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
Trial DescriptionA 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 NCT02968004
Disease/ConditionPediatric Growth Hormone Deficiency
Principal InvestigatorPing Zhou
Trial DescriptionA 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) NCT05704738
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
Trial DescriptionA 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 NCT04594369
Disease/ConditionNon-Cystic Fibrosis Bronchiectasis
Principal InvestigatorDivya Reddy
Trial DescriptionA 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 NCT05156398
Disease/ConditionMigraine
Principal InvestigatorJelena Pavlovic
Trial DescriptionA 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) NCT03619850
Disease/ConditionChronic Kidney Disease; Iron Deficiency Anemia
Principal InvestigatorAnna Zolotnitskaya
Trial DescriptionA 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 NCT04175613
Disease/ConditionPsoriasis
Principal InvestigatorJulia Gittler
Trial DescriptionA 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) NCT05601882
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
Trial DescriptionA 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 IINCT03628209
Disease/ConditionOsteosarcoma
Osteosarcoma in Children
Osteosarcoma Recurrent
Sarcoma
Principal InvestigatorDaniel Weiser
Trial DescriptionA 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) NCT04299464
Disease/ConditionAutism Spectrum Disorder (ASD)
Principal InvestigatorEric Hollander
Trial DescriptionA Phase III Randomized Study of Adjuvant Ipilimumab Anti-CTLA4 Therapy Versus High-Dose Interferon a-2b for Resected High-Risk Melanoma NCT01274338
Disease/ConditionMelanoma 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
Principal InvestigatorJoseph Sparano
Trial DescriptionA Phase III Randomized Trial for Newly Diagnosed High Risk B-Lymphoblastic Leukemia (B-ALL) Including a Stratum Evaluating Dasatinib (NSC#732517) in Patients With Ph-like Tyrosine Kinase Inhibitor (TKI) Sensitive Mutations NCT02883049
Disease/ConditionB Acute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia, BCR-ABL1-Like
Central Nervous System Leukemia
Testicular Leukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial for Patients With De Novo AML Using Bortezomib and Sorafenib (NSC# 681239, NSC# 724772) for Patients With High Allelic Ratio FLT3/ITD NCT01371981
Disease/ConditionAcute Myeloid Leukemia
Leukemia Cutis
Myeloid Neoplasm
Myeloid Sarcoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial Investigating Bortezomib (NSC# 681239) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy) NCT02112916
Disease/ConditionAdult T Acute Lymphoblastic Leukemia
Ann Arbor Stage II Adult Lymphoblastic Lymphoma
Ann Arbor Stage II Childhood Lymphoblastic Lymphoma
Ann Arbor Stage III Adult Lymphoblastic Lymphoma
Ann Arbor Stage III Childhood Lymphoblastic Lymphoma
Ann Arbor Stage IV Adult Lymphoblastic Lymphoma
Ann Arbor Stage IV Childhood Lymphoblastic Lymphoma
Childhood T Acute Lymphoblastic Leukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial of Adding Vincristine-Topotecan-Cyclophosphamide to Standard Chemotherapy in Initial Treatment of Non-Metastatic Ewing Sarcoma NCT01231906
Disease/ConditionLocalized Extraskeletal Ewing Sarcoma
Peripheral Primitive Neuroectodermal Tumor of Bone
Peripheral Primitive Neuroectodermal Tumor of Soft Tissues
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Study for Patients With Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid NCT02339740
Disease/ConditionAcute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III, Randomized Study of Nivolumab (Opdivo) Plus AVD or Brentuximab Vedotin (Adcetris) Plus AVD in Patients (Age >/= 12 Years) With Newly Diagnosed Advanced Stage Classical Hodgkin Lymphoma NCT03907488
Disease/ConditionAnn Arbor Stage III Hodgkin Lymphoma
Ann Arbor Stage III Lymphocyte-Depleted Classic Hodgkin Lymphoma
Ann Arbor Stage III Mixed Cellularity Classic Hodgkin Lymphoma
Ann Arbor Stage III Nodular Sclerosis Classic Hodgkin Lymphoma
Ann Arbor Stage IV Hodgkin Lymphoma
Ann Arbor Stage IV Lymphocyte-Depleted Classic Hodgkin Lymphoma
Ann Arbor Stage IV Mixed Cellularity Classic Hodgkin Lymphoma
Ann Arbor Stage IV Nodular Sclerosis Classic Hodgkin Lymphoma
Classic Hodgkin Lymphoma
Lymphocyte-Rich Classic Hodgkin Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma NCT04385277
Disease/ConditionGanglioneuroblastoma, Nodular
Neuroblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma NCT04385277
Disease/ConditionGanglioneuroblastoma, Nodular
Neuroblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations NCT05112237
Disease/ConditionCardiomyopathy
Principal InvestigatorDaphne Hsu
Trial DescriptionA Prospective Observational Study of Patients Receiving DUPIXENT® for Atopic Dermatitis NCT03428646
Disease/ConditionDermatitis, Atopic
Principal InvestigatorHolly Kanavy
Trial DescriptionA Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) NCT04174157
Disease/ConditionSpinal Muscular Atrophy (SMA)
Principal InvestigatorLESLIE DELFINER
Trial DescriptionA Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid) NCT03115086
Disease/ConditionBile Acid Synthesis Disorders
Principal InvestigatorNadia Ovchinsky
Trial DescriptionA 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 NCT03588975
Disease/ConditionChondral Defect
Osteochondritis Dissecans (OCD)
Articular Cartilage Defect
Articular Cartilage Disorder of Knee
Principal InvestigatorEric Fornari
Trial DescriptionA 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 NCT03588975
Disease/ConditionChondral Defect
Osteochondritis Dissecans (OCD)
Articular Cartilage Defect
Articular Cartilage Disorder of Knee
Principal InvestigatorEric Fornari
Trial DescriptionA 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 NCT05675410
Disease/ConditionLugano Classification Limited Stage Hodgkin Lymphoma AJCC v8
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized Phase 3 Study of Brentuximab Vedotin (SGN-35) for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma (cHL) in Children and Young Adults NCT02166463
Disease/ConditionAnn Arbor Stage IIB Hodgkin Lymphoma
Ann Arbor Stage IIIB Hodgkin Lymphoma
Ann Arbor Stage IVA Hodgkin Lymphoma
Ann Arbor Stage IVB Hodgkin Lymphoma
Childhood Hodgkin Lymphoma
Classic Hodgkin Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA 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) NCT02567435
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Rhabdomyosarcoma
Sclerosing Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized Phase 3 Trial of Nivolumab (NSC# 748726) in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma NCT04759586
Disease/ConditionPrimary Mediastinal Large B-Cell Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA 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 NCT02375204
Disease/ConditionGerm 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
Principal InvestigatorBenjamin Gartrell
Trial DescriptionA Randomized Trial of Levocarnitine Prophylaxis to Prevent Asparaginase-Associated Hepatotoxicity in Adolescents and Young Adults Receiving Acute Lymphoblastic Leukemia Therapy NCT05602194
Disease/ConditionB 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
Principal InvestigatorAlice Lee
Trial DescriptionA 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 NCT04113382
Disease/ConditionBowel Preparation
Principal InvestigatorInna Novak
Trial DescriptionA Randomized, Comparative Effectiveness Study of Staged Complete Revascularization With Percutaneous Coronary Intervention to Treat Coronary Artery Disease vs Medical Management Alone in Patients With Symptomatic Aortic Valve Stenosis Undergoing Elective Transfemoral Transcatheter Aortic Valve Replacement: The COMPLETE TAVR Study NCT04634240
Disease/ConditionAortic Stenosis
Coronary Artery Disease
Coronary Stenosis
Principal InvestigatorMark Menegus
Trial DescriptionA Randomized, Comparative Effectiveness Study of Staged Complete Revascularization With Percutaneous Coronary Intervention to Treat Coronary Artery Disease vs Medical Management Alone in Patients With Symptomatic Aortic Valve Stenosis Undergoing Elective Transfemoral Transcatheter Aortic Valve Replacement: The COMPLETE TAVR Study NCT04634240
Disease/ConditionAortic Stenosis
Coronary Artery Disease
Coronary Stenosis
Principal InvestigatorMark Menegus
Trial DescriptionA 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 NCT03978000
Disease/ConditionNecrotizing Enterocolitis
Principal InvestigatorShantanu Rastogi
Trial DescriptionA 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 NCT03978000
Disease/ConditionNecrotizing Enterocolitis
Principal InvestigatorShantanu Rastogi
Trial DescriptionA Randomized, Double-Blind, Double Dummy, Parallel Group, Multicenter Variable Length Study to Assess the Efficacy and Safety of PT010 Relative to PT009 and Symbicort® in Adult and Adolescent Participants With Inadequately Controlled Asthma NCT04609904
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 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.NCT05219617
Disease/ConditionSeizures
Lennox Gastaut Syndrome
Principal InvestigatorAlexis Boro
Trial DescriptionA 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 NCT04314375
Disease/ConditionUlcerative Colitis
Principal InvestigatorGitit Tomer
Trial DescriptionA 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 NCT03493685
Disease/ConditionFocal Segmental Glomerulosclerosis
Principal InvestigatorFrederick Kaskel
Trial DescriptionA 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) NCT04161885
Disease/ConditionAcute Myeloid Leukemia (AML)
Cancer
Principal InvestigatorIoannis Mantzaris
Trial DescriptionA RANDOMIZED, OPEN-LABEL, ACTIVE CONTROLLED, SAFETY AND DESCRIPTIVE EFFICACY STUDY IN PEDIATRIC SUBJECTS REQUIRING ANTICOAGULATION FOR THE TREATMENT OF A VENOUS THROMBOEMBOLIC EVENT NCT02464969
Disease/ConditionVenous Thromboembolism
Principal InvestigatorJennifer Davila
Trial DescriptionA RANDOMIZED, OPEN-LABEL, ACTIVE CONTROLLED, SAFETY AND DESCRIPTIVE EFFICACY STUDY IN PEDIATRIC SUBJECTS REQUIRING ANTICOAGULATION FOR THE TREATMENT OF A VENOUS THROMBOEMBOLIC EVENT NCT02464969
Disease/ConditionVenous Thromboembolism
Principal InvestigatorJennifer Davila
Trial DescriptionA 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.NCT04335643
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorTamar Rubinstein
Trial DescriptionA 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.NCT04335643
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorTamar Rubinstein
Trial DescriptionA Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors NCT04195399
Disease/ConditionDesmoid Fibromatosis
Recurrent Desmoid Fibromatosis
Unresectable Desmoid Fibromatosis
Principal InvestigatorLisa Gennarini
Trial DescriptionA Trial of Endocrine Response in Women With Invasive Lobular Breast Cancer NCT02206984
Disease/ConditionBreast Cancer
Principal InvestigatorSheldon Feldman
Trial DescriptionA Trial of Endocrine Response in Women With Invasive Lobular Breast Cancer NCT02206984
Disease/ConditionBreast Cancer
Principal InvestigatorSheldon Feldman
Trial DescriptionAn Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS) NCT04624659
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Trial DescriptionAn Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials NCT03573882
Disease/ConditionSickle Cell Disease
Principal InvestigatorCaterina Minniti
Trial DescriptionAn Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA) NCT03841357
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
Trial DescriptionAn Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor) NCT04930445
Disease/ConditionSickle Cell Disease
Principal InvestigatorHenny Billett
Trial DescriptionAn 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) NCT04185363
Disease/ConditionProgressive Familial Intrahepatic Cholestasis (PFIC)
Principal InvestigatorNadia Ovchinsky
Trial DescriptionAn 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) NCT05086315
Disease/ConditionAcute Lymphocytic Leukaemia
Acute Myeloid Leukaemia Refractory
Myelodysplastic Syndromes
Blastic Plasmacytoid Dendritic Cell Neoplasia
Principal InvestigatorIoannis Mantzaris
Trial DescriptionAn Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects With Epstein-Barr Virus-associated Diseases (EBVision) NCT04554914
Disease/ConditionEpstein-Barr Virus (EBV)-Associated Diseases
EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD)
EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD)
EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD)
EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
Solid Organ Transplant Complications
Lymphoproliferative Disorders
Allogeneic Hematopoietic Cell Transplant
Stem Cell Transplant Complications
EBV+ Sarcomas
Leiomyosarcoma
Principal InvestigatorDavid Loeb
Trial DescriptionAn Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects With Epstein-Barr Virus-associated Diseases (EBVision) NCT04554914
Disease/ConditionEpstein-Barr Virus (EBV)-Associated Diseases
EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD)
EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD)
EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD)
EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
Solid Organ Transplant Complications
Lymphoproliferative Disorders
Allogeneic Hematopoietic Cell Transplant
Stem Cell Transplant Complications
EBV+ Sarcomas
Leiomyosarcoma
Principal InvestigatorDavid Loeb
Trial DescriptionAssessment of CMV-specific T Cell Responses by Flow Cytometry With Intracellular Cytokine Staining to Predict CMV Infection Risk in Pediatric Solid Organ Transplant Recipients NCT03924219
Disease/ConditionHeart Transplant Infection
Kidney Transplant Infection
Liver Transplant Infection
CMV
Principal InvestigatorBetsy Herold
Trial DescriptionCancer Moonshot Biobank Research Protocol NCT04314401
Disease/ConditionAcute 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
Principal InvestigatorBalazs Halmos
Trial DescriptionCannabidivarin (CBDV) vs. Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS) NCT03848481
Disease/ConditionPrader-Willi Syndrome
Principal InvestigatorEric Hollander
Trial DescriptionCannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD) - Phase 2, 12-week double-blind, randomized, placebo-controlled trialNCT03202303
Disease/ConditionAutism Spectrum Disorder
Principal InvestigatorEric Hollander
Trial DescriptionChronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors) NCT05702645
Disease/ConditionB Acute Lymphoblastic Leukemia Associated With Down Syndrome
Down Syndrome
Myeloid Leukemia Associated With Down Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionChronic Kidney Disease in Children Prospective Cohort Study (CKiD) NCT00327860
Disease/ConditionChronic Kidney Disease
Principal InvestigatorFrederick Kaskel
Trial DescriptionComparison of Conventional Non-Invasive Ventilation and Neurally-Adjusted Ventilatory Assistance (NAVA) Non-Invasive Ventilation for the Treatment of Bronchiolitis - Non-blinded, randomized clinical trialNCT06053684
Disease/ConditionBronchiolitis
Principal InvestigatorJacqueline Weingarten-Arams
Trial DescriptionCOVID-19 Vaccine Response in People Living With Sickle Cell Disease NCT05139992
Disease/ConditionSickle Cell Disease
COVID-19
Principal InvestigatorDeepa Manwani
Trial DescriptionCOVID-19 Vaccine Response in People Living With Sickle Cell Disease NCT05139992
Disease/ConditionSickle Cell Disease
COVID-19
Principal InvestigatorDeepa Manwani
Trial DescriptionDocumentation and Delivery of Guideline-Consistent Treatment in Adolescent and Young Adult (AYA) Acute Lymphoblastic Leukemia (ALL) NCT03204916
Disease/ConditionAcute Lymphoblastic Leukemia
Principal InvestigatorDavid Loeb
Trial DescriptionEffect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial - This is a genotype stratified, double-blind, randomized, placebo-controlled, parallel-group, phase IV clinical trialNCT03694158
Disease/ConditionAsthma
Principal InvestigatorSunit Jariwala
Trial DescriptionEROS: Engendering Reproductive Health Within Oncologic Survivorship NCT01806129
Disease/ConditionMalignant Neoplasm
Principal InvestigatorDella Makower
Trial DescriptionEstablishing Continuous Cell Lines and Xenografts From Pediatric Cancers for Biological and Pre-Clinical Therapeutic Studies NCT00898755
Disease/ConditionAcute Lymphoblastic Leukemia
Acute Myeloid Leukemia
Central Nervous System Neoplasm
Ewing Sarcoma
Germ Cell Tumor
Leukemia
Lymphoma
Malignant Neoplasm
Neuroblastoma
Osteosarcoma
Retinoblastoma
Rhabdoid Tumor
Rhabdomyosarcoma
Soft Tissue Sarcoma
Principal InvestigatorPeter Cole
Trial DescriptionEvaluation of the Asthma Management Program to Promote Activity for Students in Schools (Asthma-PASS) NCT04576442
Disease/ConditionAsthma in Children
Principal InvestigatorMarina Reznik
Trial DescriptionEvaluation of the Asthma Management Program to Promote Activity for Students in Schools (Asthma-PASS) NCT04576442
Disease/ConditionAsthma in Children
Principal InvestigatorMarina Reznik
Trial DescriptionEvolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma NCT04388839
Disease/ConditionRhabdomyosarcoma
Principal InvestigatorAlice Lee
Trial DescriptionGenetic Modifiers of 22q11.2 Deletion Syndrome NCT00556530
Disease/ConditionDiGeorge Syndrome
22q11.2 Deletion Syndrome
Principal InvestigatorBernice Morrow
Trial DescriptionImplementing Models for Mechanical Circulatory Support Presurgical Assessment in Congenital Heart Disease Treatment NCT03891160
Disease/ConditionCongenital Heart Disease
Principal InvestigatorOmar Saeed
Trial DescriptionImplementing Models for Mechanical Circulatory Support Presurgical Assessment in Congenital Heart Disease Treatment NCT03891160
Disease/ConditionCongenital Heart Disease
Principal InvestigatorOmar Saeed
Trial DescriptionImproving Patient-Centered Communication in Breast Cancer: A RCT of a Shared Decision Engagement System (SHARES) NCT04549571
Disease/ConditionAnatomic 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
Principal InvestigatorSheldon Feldman
Trial DescriptionInternational Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones NCT03007147
Disease/ConditionAcute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia
Mixed Phenotype Acute Leukemia
T Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionInternational Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones NCT03007147
Disease/ConditionAcute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia
Mixed Phenotype Acute Leukemia
T Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionJ-Valve TF Early Feasibility Study NCT06034028
Disease/ConditionAortic Valve Disease
Aortic Valve Insufficiency
Aortic Regurgitation
Principal InvestigatorMohamed Azeem Latib
Trial DescriptionLarotrectinib (LOXO-101, NSC# 788607) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias NCT03834961
Disease/ConditionCentral Nervous System Neoplasm
Infantile Fibrosarcoma
Recurrent Acute Leukemia
Refractory Acute Leukemia
Solid Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionLongitudinal Assessment of Ovarian Reserve in Adolescents With Lymphoma NCT01793233
Disease/ConditionAnaplastic Large Cell Lymphoma
Burkitt Lymphoma
Diffuse Large B-Cell Lymphoma
Hodgkin Lymphoma
Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionLong-Term Follow-Up (LTFU) of Participants Treated With GSK Adoptive Cell Therapies NCT03391778
Disease/ConditionNeoplasms
Principal InvestigatorDavid Loeb
Trial DescriptionLong-Term Follow-Up (LTFU) of Participants Treated With GSK Adoptive Cell Therapies NCT03391778
Disease/ConditionNeoplasms
Principal InvestigatorDavid Loeb
Trial DescriptionMaster 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) NCT03967223
Disease/ConditionNeoplasms
Principal InvestigatorDavid Loeb
Trial DescriptionMatched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome NCT02670525
Disease/ConditionRecurrent, Refractory, or High Risk Leukemias
Matched Targeted Therapy
Principal InvestigatorLisa Gennarini
Trial DescriptionMetformin 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).NCT04758000
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Principal Investigator
Trial DescriptionMOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study NCT04023019
Disease/ConditionHemophilia A
Principal InvestigatorWilliam Mitchell
Trial DescriptionMolecular Analysis for Combination Therapy Choice (ComboMATCH) NCT05564377
Disease/ConditionAdvanced 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
Principal InvestigatorBalazs Halmos
Trial DescriptionMolecular and Genomic Profiling of Head and Neck Tumors to Explore Biomarkers Associated With Prognosis and Response to Therapy NCT00200486
Disease/ConditionHead and Neck Neoplasms
Principal InvestigatorRichard Smith
Trial DescriptionMulti-Center African-American Inflammatory Bowel Disease Study (MAAIS) NCT01169194
Disease/ConditionInflammatory Bowel Disease
Principal InvestigatorRuby Greywoode
Trial DescriptionMulticenter 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 NCT02520713
Disease/ConditionPediatric Solid Tumor
Principal InvestigatorDaniel Weiser
Trial DescriptionMulticenter 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 NCT02520713
Disease/ConditionPediatric Solid Tumor
Principal InvestigatorDaniel Weiser
Trial DescriptionMulticenter, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Subjects With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure of Rituximab or Rituximab and Chemotherapy NCT03394365
Disease/ConditionEpstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
Solid Organ Transplant Complications
Lymphoproliferative Disorders
Allogeneic Hematopoietic Cell Transplant
Stem Cell Transplant Complications
Principal InvestigatorIoannis Mantzaris
Trial DescriptionMulticenter, 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).NCT05755035
Disease/ConditionPrimary Immunodeficiency Diseases (PID)
Principal InvestigatorArye Rubinstein
Trial DescriptionMulti-Institution Registry of SRS/SBRT Procedures NCT01885299
Disease/ConditionNeoplasms
Arteriovenous Malformation of Central Nervous System
Trigeminal Neuralgia
Principal InvestigatorMadhur Garg
Trial DescriptionNational Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children With Complex Congenital Heart Disease NCT02852031
Disease/ConditionHypoplastic Left Heart Syndrome (HLHS)
Principal InvestigatorNicole Sutton
Trial DescriptionNatural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections NCT00018044
Disease/ConditionMycobacterium Infections
Principal InvestigatorSteven Porcelli
Trial DescriptionNatural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections NCT00018044
Disease/ConditionMycobacterium Infections
Principal InvestigatorSteven Porcelli
Trial DescriptionNCI COVID-19 in Cancer Patients Study (NCCAPS): A Longitudinal Natural History Study NCT04387656
Disease/ConditionCOVID-19 Infection
Hematopoietic and Lymphatic System Neoplasm
Malignant Solid Neoplasm
Metastatic Malignant Solid Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations NCT04195555
Disease/ConditionRecurrent 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
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Erdafitinib in Patients With Tumors Harboring FGFR1/2/3/4 Alterations NCT03210714
Disease/ConditionAdvanced 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
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-101 (Larotrectinib) in Patients With Tumors Harboring Actionable NTRK Fusions NCT03213704
Disease/ConditionAdvanced 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
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-292 in Patients With Tumors Harboring RET Gene Alterations NCT04320888
Disease/ConditionHematopoietic 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
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Ensartinib in Patients With Tumors Harboring ALK or ROS1 Genomic Alterations NCT03213652
Disease/ConditionAdvanced 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
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice)- Phase 2 Subprotocol of LY3023414 in Patients With Solid Tumors NCT03213678
Disease/ConditionAdvanced 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
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Tipifarnib in Patients With Tumors Harboring HRAS Genomic Alterations NCT04284774
Disease/ConditionMalignant 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
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol NCT03155620
Disease/ConditionAdvanced Malignant Solid Neoplasm
Ann Arbor Stage III Non-Hodgkin Lymphoma
Ann Arbor Stage IV Non-Hodgkin Lymphoma
Histiocytic Sarcoma
Juvenile Xanthogranuloma
Langerhans Cell Histiocytosis
Malignant Glioma
Recurrent Childhood Rhabdomyosarcoma
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 Rhabdoid Tumor
Recurrent Soft Tissue Sarcoma
Refractory Ewing Sarcoma
Refractory Glioma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
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
Principal InvestigatorAlice Lee
Trial DescriptionNephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network NCT01209000
Disease/ConditionMinimal Change Disease (MCD)
Membranous Nephropathy
Glomerulosclerosis, Focal Segmental
Principal InvestigatorFrederick Kaskel
Trial DescriptionNephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network NCT01209000
Disease/ConditionMinimal Change Disease (MCD)
Membranous Nephropathy
Glomerulosclerosis, Focal Segmental
Principal InvestigatorFrederick Kaskel
Trial DescriptionNeuroblastoma Biology Studies NCT00904241
Disease/ConditionGanglioneuroblastoma
Localized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Regional Neuroblastoma
Stage 4 Neuroblastoma
Stage 4S Neuroblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionNeurodevelopmental Outcome of Normoglycemic Versus Hypoglycemic Neonates at Risk for Hypoglycemia NCT04875624
Disease/ConditionNeonatal Hypoglycemia
Neurodevelopmental Disorders
Principal InvestigatorMariam Susan Latuga
Trial DescriptionNeuropsychological, Social, Emotional, and Behavioral Outcomes in Children With Cancer NCT00772200
Disease/ConditionChildhood Malignant Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionNMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO) NCT02679144
Disease/ConditionNeuroblastoma
Principal InvestigatorDaniel Weiser
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry NCT02418442
Disease/ConditionRheumatic Joint Disease
Principal InvestigatorDawn Wahezi
Trial DescriptionONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study NCT05580562
Disease/ConditionH3 K27M
Glioma
Principal InvestigatorAdilia Hormigo
Trial DescriptionOptimize PRO Transcatheter Aortic Valve Replacement Post Market Study NCT04091048
Disease/ConditionSymptomatic Aortic Stenosis
Principal InvestigatorMohamed Azeem Latib
Trial DescriptionOptimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure? - randomized non blinded studyNCT04479735
Disease/ConditionPain, Procedural
Principal InvestigatorCourtney Mcnamara
Trial DescriptionOutcomes of Neonatal Acute Kidney Injury and Prematurity: An Observational Study and Einstein-Montefiore Neonatal Acute Kidney Injury Clinical Registry and Biorepository NCT02375854
Disease/ConditionAcute Kidney Injury
Chronic Kidney Disease
Principal InvestigatorMamta Fuloria
Trial DescriptionPazopanib Neoadjuvant Trial in Non-Rhabdomyosarcoma Soft Tissue Sarcomas (PAZNTIS): A Phase II/III Randomized Trial of Preoperative Chemoradiation or Preoperative Radiation Plus or Minus Pazopanib (NSC# 737754) NCT02180867
Disease/ConditionAlveolar Soft Part Sarcoma
Angiomatoid Fibrous Histiocytoma
Atypical Fibroxanthoma
Clear Cell Sarcoma of Soft Tissue
Epithelioid Malignant Peripheral Nerve Sheath Tumor
Epithelioid Sarcoma
Extraskeletal Myxoid Chondrosarcoma
Extraskeletal Osteosarcoma
Fibrohistiocytic Neoplasm
Fibrosarcoma
Inflammatory Myofibroblastic Tumor
Intimal Sarcoma
Leiomyosarcoma
Liposarcoma
Liver Embryonal Sarcoma
Low Grade Fibromyxoid Sarcoma
Low Grade Myofibroblastic Sarcoma
Malignant Cutaneous Granular Cell Tumor
Malignant Peripheral Nerve Sheath Tumor
Malignant Triton Tumor
Mesenchymal Chondrosarcoma
Myxofibrosarcoma
Myxoid Chondrosarcoma
Myxoinflammatory Fibroblastic Sarcoma
Nerve Sheath Neoplasm
PEComa
Pericytic Neoplasm
Plexiform Fibrohistiocytic Tumor
Sclerosing Epithelioid Fibrosarcoma
Skin Glomus Tumor
Stage IB Soft Tissue Sarcoma AJCC v7
Stage IIB Soft Tissue Sarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Synovial Sarcoma
Undifferentiated High Grade Pleomorphic Sarcoma of Bone
Principal InvestigatorLisa Gennarini
Trial DescriptionPediatric Hepatic Malignancy International Therapeutic Trial (PHITT) NCT03533582
Disease/ConditionChildhood Hepatocellular Carcinoma
Childhood Malignant Liver Neoplasm
Fibrolamellar Carcinoma
Hepatoblastoma
Hepatocellular Malignant Neoplasm, Not Otherwise Specified
Principal InvestigatorAlice Lee
Trial DescriptionPHASE 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).NCT03709680
Disease/ConditionEwing Sarcoma
Solid Tumors
Rhabdoid Tumor
Rhabdomyosarcoma
Neuroblastoma
Medulloblastoma
Diffuse Intrinsic Pontine Glioma
Principal InvestigatorAlice Lee
Trial DescriptionPHASE 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).NCT03709680
Disease/ConditionEwing Sarcoma
Solid Tumors
Rhabdoid Tumor
Rhabdomyosarcoma
Neuroblastoma
Medulloblastoma
Diffuse Intrinsic Pontine Glioma
Principal InvestigatorAlice Lee
Trial DescriptionPhase 1-2 Trial of Gamunex (Intravenous Gammaglobulin) for Sickle Cell Acute Pain NCT01757418
Disease/ConditionSickle Cell Disease
Pain
Principal InvestigatorDeepa Manwani
Trial DescriptionPhase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis NCT05828069
Disease/ConditionRecurrent Langerhans Cell Histiocytosis
Refractory Langerhans Cell Histiocytosis
Principal InvestigatorAlice Lee
Trial DescriptionPhase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome NCT03197662
Disease/ConditionPrader-Willi Syndrome
Hyperphagia
Principal InvestigatorEric Hollander
Trial DescriptionPhase 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 NCT03301168
Disease/ConditionAcute Lymphoblastic Leukemia
Leukemia, Acute Myeloid (AML), Child
Lymphoma, Non-Hodgkin
Myelodysplastic Syndromes
Primary Immune Deficiency Disorder
Osteopetrosis
Cytopenia
Hemoglobinopathy in Children
Anemia, Aplastic
Principal InvestigatorMichelle Lee
Trial DescriptionPhase II Study of Nab-Paclitaxel in Combination With Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults NCT02945800
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Rhabdomyosarcoma
Soft Tissue Sarcoma
Principal InvestigatorDaniel Weiser
Trial DescriptionPhase II Study of Nab-Paclitaxel in Combination With Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults NCT02945800
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Rhabdomyosarcoma
Soft Tissue Sarcoma
Principal InvestigatorDaniel Weiser
Trial DescriptionPhase 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.NCT03386539
Disease/ConditionPediatric Heart Transplantation
Immunosuppression
Chronic Kidney Diseases
Cardiac Allograft Vasculopathy
Heart Transplant Failure and Rejection
Post-transplant Lymphoproliferative Disorder
Heart Transplant Infection
Principal InvestigatorDaphne Hsu
Trial DescriptionPhase III Randomized Trial of Post-Radiation Chemotherapy in Patients With Newly Diagnosed Ependymoma Ages 1 to 21 Years NCT01096368
Disease/ConditionAnaplastic Ependymoma
Brain Ependymoma
Cellular Ependymoma
Clear Cell Ependymoma
Ependymoma
Papillary Ependymoma
Principal InvestigatorAlice Lee
Trial DescriptionPhosphate Binder Therapy and Chronic Kidney Disease in Children NCT04741646
Disease/ConditionChronic Kidney Diseases
Principal InvestigatorFrederick Kaskel
Trial DescriptionPromoting Asthma Guidelines and Management Through Technology-Based Intervention and Care Coordination - enhanced Usual Care and Intervention sitesNCT03066596
Disease/ConditionAsthma Childhood
Principal InvestigatorMarina Reznik
Trial DescriptionProspective Cohort Study to Evaluate Immunologic Response Following COVID-19 Vaccination in Children, Adolescents and Young Adults With Cancer NCT05228275
Disease/ConditionCOVID-19 Infection
Hematopoietic and Lymphatic System Neoplasm
Malignant Solid Neoplasm
Principal InvestigatorAlice Lee
Trial DescriptionProtocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries NCT01166009
Disease/ConditionAutologous Stem Cell Transplantation
Allogeneic Stem Cell Transplantation
Solid Tumors
Blood Cancers
CAR-T
Gene Therapy
Non-malignant Disease
Principal InvestigatorMichelle Lee
Trial DescriptionRandomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial NCT03107871
Disease/ConditionCmv Congenital
CMV
Congenital Cmv
SNHL
Sensorineural Hearing Loss
Principal InvestigatorMichel Nassar
Trial DescriptionRandomized 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 NCT02306161
Disease/ConditionMetastatic 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
Principal InvestigatorAlice Lee
Trial DescriptionRandomized Study of Intravenous Calaspargase Pegol (SC-PEG Asparaginase) and Intravenous Oncaspar in Children and Adolescents With Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma NCT01574274
Disease/ConditionAcute Lymphoblastic Leukemia
Lymphoblastic Lymphoma
Principal InvestigatorLisa Gennarini
Trial DescriptionREal-World Analyses of Stroke - Thrombus Occlusion REtrieval NCT04451525
Disease/ConditionAcute Ischemic Stroke
Vessel Occlusion
Stroke
Ischemic
Principal InvestigatorNeil Haranhalli
Trial DescriptionRegistry of Asthma Patients Initiating DUPIXENT® (RAPID) NCT04287621
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
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
Trial DescriptionRisk-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) NCT02927769
Disease/ConditionHodgkin Disease
Principal InvestigatorLisa Gennarini
Trial DescriptionRisk-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) NCT02927769
Disease/ConditionHodgkin Disease
Principal InvestigatorLisa Gennarini
Trial DescriptionRisk-Stratified Randomized Phase III Testing of Blinatumomab (NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL) NCT02101853
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionRisk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome NCT02521493
Disease/ConditionAcute Myeloid Leukemia
Down Syndrome
Myelodysplastic Syndrome
Myeloid Leukemia Associated With Down Syndrome
Myeloproliferative Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionSafety and Effectiveness of Balloon-Expandable Bioprosthetic SAPIEN X4 Transcatheter Heart Valve NCT05172960
Disease/ConditionAortic Stenosis, Severe
Principal InvestigatorMohamed Azeem Latib
Trial DescriptionSickle Cell Disease (SCD) Biochip': Towards a Simple and Reliable Way to Monitor Sickle Cell Disease NCT02824471
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Trial DescriptionSimple Bone Cysts in Kids (SBoCK) NCT02193841
Disease/ConditionBone Cyst
Principal InvestigatorJacob Schulz
Trial DescriptionStudy for the Prediction of Active Rejection in Organs Using Donor-derived Cell-free DNA Detection NCT03984747
Disease/ConditionKidney Transplant Rejection
Allograft Rejection
Kidney Transplant; Complications
Principal InvestigatorNicole Hayde
Trial DescriptionStudy for the Prediction of Active Rejection in Organs Using Donor-derived Cell-free DNA Detection NCT03984747
Disease/ConditionKidney Transplant Rejection
Allograft Rejection
Kidney Transplant; Complications
Principal InvestigatorNicole Hayde
Trial DescriptionSurveillance HeartCare® Outcomes Registry NCT03695601
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionThe Collection of Research Samples and/or Data for Repository From Related or Unrelated Hematopoietic Stem Cell Transplantation Recipients for the National Marrow Donor Program NCT00495300
Disease/ConditionBone Marrow Transplantation
Principal InvestigatorIra Braunschweig
Trial DescriptionThe Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study NCT02402244
Disease/ConditionCarcinoma 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
Principal InvestigatorAlice Lee
Trial DescriptionThe Tether™ - Vertebral Body Tethering System Post Approval Study NCT04505579
Disease/ConditionScoliosis Idiopathic
Principal InvestigatorJaime Gomez
Trial DescriptionThe Tether™ - Vertebral Body Tethering System Post Approval Study NCT04505579
Disease/ConditionScoliosis Idiopathic
Principal InvestigatorJaime Gomez
Trial DescriptionTransvenous Approach for the Treatment of Cerebral Arteriovenous Malformations (TATAM): A Randomized Controlled Trial and Registry NCT03691870
Disease/ConditionArteriovenous Malformations, Cerebral
Unruptured Brain Arteriovenous Malformation
Ruptured Brain Arteriovenous Malformation
Principal InvestigatorDavid Altschul
Trial DescriptionTreatment of Children With All Stages of Hepatoblastoma With Temsirolimus (NSC#683864) Added to High Risk Stratum Treatment NCT00980460
Disease/ConditionPRETEXT I Hepatoblastoma
PRETEXT II Hepatoblastoma
PRETEXT III Hepatoblastoma
PRETEXT IV Hepatoblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionTreatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents NCT03020030
Disease/ConditionAcute Lymphoblastic Leukemia, Pediatric
Principal InvestigatorLisa Gennarini
Trial DescriptionTreatment of Patients With Newly Diagnosed Standard Risk B-Lymphoblastic Leukemia (B-ALL) or Localized B-Lineage Lymphoblastic Lymphoma (B-LLy) NCT01190930
Disease/ConditionAcute 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
Principal InvestigatorLisa Gennarini
Trial DescriptionTrial of Infant Probiotic Supplementation to Prevent Asthma NCT00113659
Disease/ConditionAsthma
Principal InvestigatorMichael Cabana
Trial DescriptionTrialNet Pathway to Prevention of T1D NCT00097292
Disease/ConditionDiabetes Mellitus, Type 1
Principal InvestigatorLisa Underland
Trial DescriptionTrialNet Pathway to Prevention of T1D NCT00097292
Disease/ConditionDiabetes Mellitus, Type 1
Principal InvestigatorLisa Underland
Trial DescriptionTrifecta-Heart cfDNA-MMDX Study: Comparing the DD-cfDNA Test to MMDx Microarray Test and Central HLA Antibody Test NCT04707872
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionTumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial NCT04589845
Disease/ConditionSolid Tumors
Principal InvestigatorHaiying Cheng
Trial DescriptionUmbrella Long-Term Follow-Up Protocol NCT00736749
Disease/ConditionHematopoietic Cell Transplantation Recipient
Leukemia
Solid Tumor
Principal InvestigatorAlice Lee
Trial DescriptionUtilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients With Non-High-Risk Neuroblastoma NCT02176967
Disease/ConditionGanglioneuroblastoma
Localized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Neuroblastoma
Principal InvestigatorAlice Lee
Trial DescriptionViral Infection and Respiratory Illness Universal Study[VIRUS]: COVID-19 Registry and Validation of C2D2 (Critical Care Data Dictionary) NCT04323787
Disease/ConditionCoronavirus
Principal InvestigatorJen-Ting Chen