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 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 NCT04718389
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease NCT03251118
Disease/ConditionInflammatory Bowel Diseases
Crohn's Disease
Ulcerative Colitis
Indeterminate Colitis
Principal InvestigatorGitit Tomer
Trial DescriptionA 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease NCT03251118
Disease/ConditionInflammatory Bowel Diseases
Crohn's Disease
Ulcerative Colitis
Indeterminate Colitis
Principal InvestigatorGitit Tomer
Trial DescriptionA Comprehensive Approach to Improve Medication Adherence in Pediatric ALL NCT01503632
Disease/ConditionAcute Lymphoblastic Leukemia
Principal InvestigatorLisa Gennarini
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 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 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 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, 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 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 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 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 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 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-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 NCT01649765
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorDawn Wahezi
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-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 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 NCT04223752
Disease/ConditionNosocomial Pneumonia
Principal InvestigatorMichelle Collins-Ogle
Trial DescriptionA 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 NCT04223752
Disease/ConditionNosocomial Pneumonia
Principal InvestigatorMichelle Collins-Ogle
Trial DescriptionA 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) NCT02625480
Disease/ConditionRelapsed/Refractory B-precursor Acute Lymphoblastic Leukemia
Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma
Principal InvestigatorEllen Fraint
Trial DescriptionA 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) NCT02625480
Disease/ConditionRelapsed/Refractory B-precursor Acute Lymphoblastic Leukemia
Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma
Principal InvestigatorEllen Fraint
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 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 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 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 SNDX-5613 in Combination With Chemotherapy for Patients With Relapsed or Refractory KMT2A-Rearranged Infant Leukemia NCT05761171
Disease/ConditionRecurrent Acute Leukemia of Ambiguous Lineage
Recurrent Acute Lymphoblastic Leukemia
Recurrent Acute Myeloid Leukemia Due to Lineage Switch From Acute Leukemia of Ambiguous Lineage
Recurrent Acute Myeloid Leukemia Due to Lineage Switch From B Acute Lymphoblastic Leukemia, KMT2A-Rearranged
Recurrent Acute Myeloid Leukemia Due to Lineage Switch From Mixed Phenotype Acute Leukemia
Recurrent Mixed Phenotype Acute Leukemia
Refractory Acute Leukemia of Ambiguous Lineage
Refractory Acute Lymphoblastic Leukemia
Refractory Acute Myeloid Leukemia Due to Lineage Switch From Acute Leukemia of Ambiguous Lineage
Refractory Acute Myeloid Leukemia Due to Lineage Switch From B Acute Lymphoblastic Leukemia, KMT2A-Rearranged
Refractory Acute Myeloid Leukemia Due to Lineage Switch From Mixed Phenotype Acute Leukemia
Refractory Mixed Phenotype Acute Leukemia
Principal InvestigatorAlice Lee
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 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 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 NCT04684368
Disease/ConditionCentral 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
Principal InvestigatorLisa Gennarini
Trial DescriptionA 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 NCT03474965
Disease/ConditionSickle Cell Disease (SCD)
Principal InvestigatorDeepa Manwani
Trial DescriptionA 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 NCT03474965
Disease/ConditionSickle Cell Disease (SCD)
Principal InvestigatorDeepa Manwani
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 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 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 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 InvestigatorAlice Lee
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 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 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 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, 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, 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, Open-label, Long-term Safety Study of Oral Linaclotide Administered to Pediatric Participants With Functional Constipation (FC) or Irritable Bowel Syndrome With Constipation (IBS-C) NCT04166058
Disease/ConditionIrritable Bowel Syndrome With Constipation
Functional Constipation
Principal InvestigatorJohn Thompson
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 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, 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 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, 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 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 II Pilot Trial to Estimate Survival After a Non-total Body Irradiation (TBI) Based Conditioning Regimen in Patients Diagnosed With B-acute Lymphoblastic Leukemia (ALL) Who Are Pre-allogeneic Hematopoietic Cell Transplantation (HCT) Next-generation-sequence (NGS) Minimal Residual Disease (MRD) Negative NCT03509961
Disease/ConditionB-cell Acute Lymphoblastic Leukemia
Principal InvestigatorDavid Loeb
Trial DescriptionA Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors NCT01804634
Disease/ConditionRefractory and/or Relapsed Metastatic Solid Tumors
Principal InvestigatorDavid Loeb
Trial DescriptionA Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors NCT01804634
Disease/ConditionRefractory and/or Relapsed Metastatic Solid Tumors
Principal InvestigatorDavid Loeb
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 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 NCT00336024
Disease/ConditionAnaplastic Medulloblastoma
Medulloblastoma
Supratentorial Embryonal Tumor, Not Otherwise Specified
Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor
Principal InvestigatorJonathan Gill
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 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 Phase III, Randomized, Double-blind, Parallel-group, Placebo Controlled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of IV Anifrolumab in Pediatric Participants 5 to < 18 Years of Age With Moderate to Severe Active Systemic Lupus Erythematosus (SLE) While on Background Standard of Care Therapy NCT05835310
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorDawn Wahezi
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 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 Observational Study of Patients Receiving DUPIXENT® for Atopic Dermatitis NCT03428646
Disease/ConditionDermatitis, Atopic
Principal InvestigatorHolly Kanavy
Trial DescriptionA Prospective, Multi-center Registry for Patients With Short Bowel Syndrome NCT01990040
Disease/ConditionShort Bowel Syndrome
Principal InvestigatorJohn Thompson
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 Controlled Trial of Women Involved in Supporting Health (WISH), a Peer-Led Intervention to Improve Postpartum Retention in HIV Care NCT04168008
Disease/ConditionHIV/AIDS
Principal InvestigatorRodney Wright
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 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) NCT04994132
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Metastatic Embryonal Rhabdomyosarcoma
Metastatic Rhabdomyosarcoma
Solid Alveolar Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
Spindle Cell/Sclerosing Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Trial DescriptionA 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) NCT04994132
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Metastatic Embryonal Rhabdomyosarcoma
Metastatic Rhabdomyosarcoma
Solid Alveolar Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
Spindle Cell/Sclerosing Rhabdomyosarcoma
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 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, 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, 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 Evaluation of the Pharmacokinetics, Safety, and Antiviral Efficacy of Tenofovir Alafenamide (TAF) in Children and Adolescent Subjects With Chronic Hepatitis B Virus Infection NCT02932150
Disease/ConditionChronic Hepatitis B
Principal InvestigatorDebra Pan
Trial DescriptionA 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 NCT02932150
Disease/ConditionChronic Hepatitis B
Principal InvestigatorDebra Pan
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, 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, 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 Remotely Delivered CBT Intervention for Youth With cSLE: A Multi-site Patient-engaged Investigation NCT04335643
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorTamar Rubinstein
Trial DescriptionA Remotely Delivered CBT Intervention for Youth With cSLE: A Multi-site Patient-engaged Investigation 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 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 NCT00085735
Disease/ConditionUntreated Childhood Medulloblastoma
Principal InvestigatorPeter Cole
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 DescriptionACURATE IDE: Transcatheter Replacement of Stenotic Aortic Valve Through Implantation of ACURATE in Subjects InDicatEd for TAVR NCT03735667
Disease/ConditionAortic Stenosis
Principal InvestigatorMohamed Azeem Latib
Trial DescriptionACURATE IDE: Transcatheter Replacement of Stenotic Aortic Valve Through Implantation of ACURATE in Subjects InDicatEd for TAVR NCT03735667
Disease/ConditionAortic Stenosis
Principal InvestigatorMohamed Azeem Latib
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 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 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, 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 of CM-AT for the Treatment of Children With Autism With All Levels of Fecal Chymotrypsin NCT02649959
Disease/ConditionAutism
Principal InvestigatorEric Hollander
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 Feasibility Study to Assess the Safety and Pharmacokinetics of Enasidenib in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (R/R-AML) With an Isocitrate Dehydrogenase-2 (IDH2) Mutation NCT04203316
Disease/ConditionRecurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionAn Open-Label, Single-Arm, Multi-Center, Phase 3 Study on the Efficacy, Safety, and Pharmacokinetics of Debio 4326, a Triptorelin 12-month Formulation, in Pediatric Participants Who Are Receiving Gonadotropin-Releasing Hormone Agonist Therapy for Central Precocious Puberty NCT06129539
Disease/ConditionCentral Precocious Puberty
Principal InvestigatorLaurie Cohen
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 DescriptionAPOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO) NCT03615235
Disease/ConditionKidney Diseases
Kidney Failure
Kidney Disease, Chronic
Principal InvestigatorEnver Akalin
Trial DescriptionAPOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO) NCT03615235
Disease/ConditionKidney Diseases
Kidney Failure
Kidney Disease, Chronic
Principal InvestigatorEnver Akalin
Trial DescriptionCannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD) NCT03202303
Disease/ConditionAutism Spectrum Disorder
Principal InvestigatorEric Hollander
Trial DescriptionCannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD) NCT03202303
Disease/ConditionAutism Spectrum Disorder
Principal InvestigatorEric Hollander
Trial DescriptionChronic Kidney Disease in Children Prospective Cohort Study (CKiD) NCT00327860
Disease/ConditionChronic Kidney Disease
Principal InvestigatorFrederick Kaskel
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 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 NCT03694158
Disease/ConditionAsthma
Principal InvestigatorSunit Jariwala
Trial DescriptionEfficacy & Safety of Pharmacokinetically-Driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis- A Double-Blind Placebo Controlled Clinical Trial NCT05538208
Disease/ConditionLupus Nephritis
Principal InvestigatorDawn Wahezi
Trial DescriptionEfficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients NCT00392327
Disease/ConditionAnaplastic Medulloblastoma
Medulloblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionEliminating Monitor Overuse (EMO) Hybrid Effectiveness-Deimplementation Trial NCT05132322
Disease/ConditionBronchiolitis Acute Viral
Principal InvestigatorAlyssa Silver
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 DescriptionGenetic Modifiers of 22q11.2 Deletion Syndrome NCT00556530
Disease/ConditionDiGeorge Syndrome
22q11.2 Deletion Syndrome
Principal InvestigatorBernice Morrow
Trial DescriptionGenetic Modifiers of 22q11.2 Deletion Syndrome NCT00556530
Disease/ConditionDiGeorge Syndrome
22q11.2 Deletion Syndrome
Principal InvestigatorBernice Morrow
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 DescriptionJ-Valve TF Early Feasibility Study NCT06034028
Disease/ConditionAortic Valve Disease
Aortic Valve Insufficiency
Aortic Regurgitation
Principal InvestigatorMohamed Azeem Latib
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 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 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 DescriptionMetformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse NCT04758000
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Principal Investigator
Trial DescriptionMinimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease NCT03587272
Disease/ConditionSickle Cell Disease
Principal InvestigatorDavid Loeb
Trial DescriptionMinimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease NCT03587272
Disease/ConditionSickle Cell Disease
Principal InvestigatorDavid Loeb
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 DescriptionMolecular Basis of Early Childhood Obesity Programming by Intrauterine Growth Restriction NCT03402139
Disease/ConditionChildhood Obesity
Epigenetics
Principal InvestigatorMamta Fuloria
Trial DescriptionMolecular Basis of Early Childhood Obesity Programming by Intrauterine Growth Restriction NCT03402139
Disease/ConditionChildhood Obesity
Epigenetics
Principal InvestigatorMamta Fuloria
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 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 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 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 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 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 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 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-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 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 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 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)- 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 DescriptionNeuroblastoma Biology Studies NCT00904241
Disease/ConditionGanglioneuroblastoma
Localized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Regional Neuroblastoma
Stage 4 Neuroblastoma
Stage 4S Neuroblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionNeuropsychological, Social, Emotional, and Behavioral Outcomes in Children With Cancer NCT00772200
Disease/ConditionChildhood Malignant Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry NCT02418442
Disease/ConditionRheumatic Joint Disease
Principal InvestigatorDawn Wahezi
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry NCT02418442
Disease/ConditionRheumatic Joint Disease
Principal InvestigatorDawn Wahezi
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry NCT02418442
Disease/ConditionRheumatic Joint Disease
Principal InvestigatorDawn Wahezi
Trial DescriptionOptimize PRO Transcatheter Aortic Valve Replacement Post Market Study NCT04091048
Disease/ConditionSymptomatic Aortic Stenosis
Principal InvestigatorMohamed Azeem Latib
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? NCT04479735
Disease/ConditionPain, Procedural
Principal InvestigatorCourtney Mcnamara
Trial DescriptionOptimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure? NCT04479735
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 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 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 Acute Leukemia (PedAL) Screening Trial - Developing New Therapies for Relapsed Leukemias NCT04726241
Disease/ConditionAcute 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
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 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 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 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: 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 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 2 Trial of Response-Based Radiation Therapy for Patients With Localized Central Nervous System Germ Cell Tumors (CNS GCT) NCT01602666
Disease/ConditionCentral Nervous System Nongerminomatous Germ Cell Tumor
Childhood Central Nervous System Germinoma
Principal InvestigatorLisa Gennarini
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 Randomized Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue NCT00026312
Disease/ConditionLocalized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Recurrent Neuroblastoma
Regional Neuroblastoma
Stage 4 Neuroblastoma
Stage 4S Neuroblastoma
Principal InvestigatorJonathan Gill
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 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 NCT03066596
Disease/ConditionAsthma Childhood
Principal InvestigatorMarina Reznik
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 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 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 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 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 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 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 DescriptionSimple Bone Cysts in Kids (SBoCK) NCT02193841
Disease/ConditionBone Cyst
Principal InvestigatorJacob Schulz
Trial DescriptionSimple Bone Cysts in Kids (SBoCK) NCT02193841
Disease/ConditionBone Cyst
Principal InvestigatorJacob Schulz
Trial DescriptionSingle-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 NCT03465592
Disease/ConditionSarcoma
Solid Tumor, Adult
Solid Tumor, Childhood
Principal InvestigatorDavid Loeb
Trial DescriptionSingle-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 NCT03465592
Disease/ConditionSarcoma
Solid Tumor, Adult
Solid Tumor, Childhood
Principal InvestigatorDavid Loeb
Trial DescriptionStopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP) NCT03817398
Disease/ConditionChronic Phase Chronic Myeloid Leukemia, BCR-ABL1 Positive
Principal InvestigatorAlice Lee
Trial DescriptionStopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP) NCT03817398
Disease/ConditionChronic Phase Chronic Myeloid Leukemia, BCR-ABL1 Positive
Principal InvestigatorAlice Lee
Trial DescriptionStudy for the Prediction of Active Rejection in Organs Using Donor-derived Cell-free DNA Detection NCT03984747
Disease/ConditionTransplant Rejection
Allograft Rejection
Complications
Principal InvestigatorNicole Hayde
Trial DescriptionStudy for the Prediction of Active Rejection in Organs Using Donor-derived Cell-free DNA Detection NCT03984747
Disease/ConditionTransplant Rejection
Allograft Rejection
Complications
Principal InvestigatorNicole Hayde
Trial DescriptionTesting the Feasibility of a Teletherapy Plus Parent Coaching Intervention for Children With Autism NCT04832152
Disease/ConditionAutism Spectrum Disorder
Principal InvestigatorElizabeth Ridgway
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/ConditionAdrenal Gland Pheochromocytoma
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 Neoplasm
Malignant Solid Neoplasm
Melanocytic Neoplasm
Myeloproliferative Neoplasm
Neoplasm of Uncertain Malignant Potential
Neuroendocrine Neoplasm
Stromal Neoplasm
Principal InvestigatorAlice Lee
Trial DescriptionThe Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study NCT02402244
Disease/ConditionAdrenal Gland Pheochromocytoma
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 Neoplasm
Malignant Solid Neoplasm
Melanocytic Neoplasm
Myeloproliferative Neoplasm
Neoplasm of Uncertain Malignant Potential
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 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 for Patients With Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor NCT00945009
Disease/ConditionAdult 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
Principal InvestigatorLisa Gennarini
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 Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT) NCT04322318
Disease/ConditionAnaplastic Kidney Wilms Tumor
Recurrent Kidney Wilms Tumor
Stage II Kidney Wilms Tumor
Stage III Kidney Wilms Tumor
Stage IV Kidney Wilms Tumor
Principal InvestigatorAlice Lee
Trial DescriptionTumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial NCT04589845
Disease/ConditionSolid Tumors
Principal InvestigatorHaiying Cheng
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 DescriptionUS Selumetinib Registry NCT05683678
Disease/ConditionNeurofibromatosis Type 1
Plexiform Neurofibromas
Principal InvestigatorNagma Dalvi
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