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
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
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease
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
Disease/ConditionAcute Lymphoblastic Leukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA Comprehensive Approach to Improve Medication Adherence in Pediatric ALL
Disease/ConditionAcute Lymphoblastic Leukemia
Principal InvestigatorLisa Gennarini
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
Disease/ConditionBiliary Atresia
Principal InvestigatorDebra Pan
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
Disease/ConditionBiliary Atresia
Principal InvestigatorDebra Pan
Trial DescriptionA Longitudinal Observational Study of Patients Undergoing Therapy for Immune-Mediated Inflammatory Skin Conditions
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)
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
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
Disease/ConditionLennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Principal InvestigatorPuja Patel
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
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
Disease/ConditionInfusion Reactions
Principal InvestigatorDavid Loeb
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)
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
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
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
Disease/ConditionPediatric Sarcoma, Refractory
Pediatric Sarcoma, Relapsed
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 1, First-in-Human, Dose Escalation and Expansion Study to Evaluate the Safety and Tolerability of XmAb541 in Advanced Solid Tumors
Disease/ConditionOvarian Cancer
Endometrial Cancer
Germ Cell Tumor
Testicular Germ Cell Tumor
Ovarian Germ Cell Tumor
Principal InvestigatorEric Feldman
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)
Disease/ConditionRelapsed/Refractory B-precursor Acute Lymphoblastic Leukemia
Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma
Principal InvestigatorEllen Fraint
Trial DescriptionA Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Disease/ConditionAnaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Diffuse Intrinsic Pontine Glioma
Diffuse Midline Glioma, H3 K27M-Mutant
Glioblastoma
Glioblastoma, Not Otherwise Specified
Malignant Glioma
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
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
Disease/ConditionHigh Risk Neuroblastoma
Recurrent Neuroblastoma
Refractory Neuroblastoma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma
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)
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)
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
Disease/ConditionMedulloblastoma
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
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
Disease/ConditionSickle Cell Disease (SCD)
Principal InvestigatorDeepa Manwani
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
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)
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
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
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
Disease/ConditionPlexiform Neurofibroma
Neurofibromatosis Type 1 (NF1)
Principal InvestigatorNagma Dalvi
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)
Disease/ConditionArthritis, Juvenile
Principal InvestigatorDawn Wahezi
Trial DescriptionA Phase 3 Randomized Controlled Trial Comparing Open vs Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
Disease/ConditionMetastatic Malignant Neoplasm in the Lung
Metastatic Osteosarcoma
Osteosarcoma
Principal InvestigatorAlice Lee
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)
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)
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
Disease/ConditionAcute Myeloid Leukemia
Principal InvestigatorAlice Lee
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
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
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
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
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
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)
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)
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
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, 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
Disease/ConditionPre-Exposure Prophylaxis of HIV Infection
Principal InvestigatorMichelle Collins-Ogle
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
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
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
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)
Disease/ConditionImmune Thrombocytopenia
Principal InvestigatorIrina Murakhovskaya
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)
Disease/ConditionIrritable Bowel Syndrome With Constipation
Functional Constipation
Principal InvestigatorJohn Thompson
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)
Disease/ConditionIrritable Bowel Syndrome With Constipation
Functional Constipation
Principal InvestigatorJohn Thompson
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
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
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
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
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)
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
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
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)
Disease/ConditionChronic Kidney Disease; Iron Deficiency Anemia
Principal InvestigatorAnna Zolotnitskaya
Trial DescriptionA Phase I Study With an Expansion Cohort/Randomized Phase II Study of the Combinations of Ipilimumab, Nivolumab and Brentuximab Vedotin in Patients With Relapsed/Refractory Hodgkin Lymphoma
Disease/ConditionRecurrent Classic Hodgkin Lymphoma
Refractory Classic Hodgkin Lymphoma
Principal InvestigatorLisa Gennarini
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
Disease/ConditionOsteosarcoma
Osteosarcoma in Children
Osteosarcoma Recurrent
Sarcoma
Principal InvestigatorDaniel Weiser
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
Disease/ConditionOsteosarcoma
Osteosarcoma in Children
Osteosarcoma Recurrent
Sarcoma
Principal InvestigatorDaniel Weiser
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
Disease/ConditionRefractory and/or Relapsed Metastatic Solid Tumors
Principal InvestigatorDavid Loeb
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
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
Disease/ConditionAcute Myeloid Leukemia
Leukemia Cutis
Myeloid Neoplasm
Myeloid Sarcoma
Principal InvestigatorLisa Gennarini
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
Disease/ConditionAnaplastic Medulloblastoma
Medulloblastoma
Supratentorial Embryonal Tumor, Not Otherwise Specified
Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor
Principal InvestigatorJonathan Gill
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
Disease/ConditionAnaplastic Medulloblastoma
Medulloblastoma
Supratentorial Embryonal Tumor, Not Otherwise Specified
Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor
Principal InvestigatorJonathan Gill
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)
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 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
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 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
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
Disease/ConditionGanglioneuroblastoma, Nodular
Neuroblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Pilot Study to Assess Aevice Medical Device for Detection of Wheeze in Pediatric Subjects and Use for Remote Auscultation
Disease/ConditionSubject Presenting Wheeze
COPD
Asthma
Principal InvestigatorSunit Jariwala
Trial DescriptionA Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations
Disease/ConditionCardiomyopathy
Principal InvestigatorDaphne Hsu
Trial DescriptionA Prospective Phase 3 Study of Patients With Newly Diagnosed Very Low-Risk and Low-Risk Fusion Negative Rhabdomyosarcoma
Disease/ConditionEmbryonal Rhabdomyosarcoma
Fusion-Negative Alveolar Rhabdomyosarcoma
Spindle Cell/Sclerosing Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Trial DescriptionA Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
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)
Disease/ConditionBile Acid Synthesis Disorders
Principal InvestigatorNadia Ovchinsky
Trial DescriptionA Randomized Controlled Trial of Women Involved in Supporting Health (WISH), a Peer-Led Intervention to Improve Postpartum Retention in HIV Care
Disease/ConditionHIV/AIDS
Principal InvestigatorRodney Wright
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
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
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)
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Rhabdomyosarcoma
Sclerosing Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
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)
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
Disease/ConditionPrimary Mediastinal Large B-Cell Lymphoma
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)
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 Study of Dexamethasone as Adjuvant Therapy for Acute Post-traumatic Headache
Disease/ConditionPost-Traumatic Headache
Principal InvestigatorBenjamin Friedman
Trial DescriptionA Randomized Trial of Levocarnitine Prophylaxis to Prevent Asparaginase-Associated Hepatotoxicity in Adolescents and Young Adults Receiving Acute Lymphoblastic Leukemia Therapy
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
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
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
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
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
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
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
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
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
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
Disease/ConditionFocal Segmental Glomerulosclerosis
Principal InvestigatorFrederick Kaskel
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
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)
Disease/ConditionAcute Myeloid Leukemia (AML)
Cancer
Principal InvestigatorIoannis Mantzaris
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)
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
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
Disease/ConditionDesmoid Fibromatosis
Recurrent Desmoid Fibromatosis
Unresectable Desmoid Fibromatosis
Principal InvestigatorLisa Gennarini
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
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
Disease/ConditionMedulloblastoma
Principal InvestigatorPeter Cole
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
Disease/ConditionMedulloblastoma
Principal InvestigatorPeter Cole
Trial DescriptionACURATE IDE: Transcatheter Replacement of Stenotic Aortic Valve Through Implantation of ACURATE in Subjects InDicatEd for TAVR
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
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)
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
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)
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
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)
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
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)
Disease/ConditionProgressive Familial Intrahepatic Cholestasis (PFIC)
Principal InvestigatorNadia Ovchinsky
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)
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)
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)
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)
Disease/ConditionKidney Diseases
Kidney Failure
Kidney Disease, Chronic
Principal InvestigatorEnver Akalin
Trial DescriptionAssessing the Effectiveness of Pictographs for Preventing Wrong-Patient Errors in Neonatal Intensive Care Units: A Randomized Controlled Trial
Disease/ConditionMedical Errors
Electronic Medical Records
Principal InvestigatorKatie Forman
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
Disease/ConditionHeart Transplant Infection
Kidney Transplant Infection
Liver Transplant Infection
CMV
Principal InvestigatorBetsy Herold
Trial DescriptionCancer Moonshot Biobank Research Protocol
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 With Autism Spectrum Disorder (ASD)
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)
Disease/ConditionB Acute Lymphoblastic Leukemia Associated With Down Syndrome
Down Syndrome
Myeloid Leukemia Associated With Down Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionComparison of Conventional Non-Invasive Ventilation and Neurally-Adjusted Ventilatory Assistance (NAVA) Non-Invasive Ventilation for the Treatment of Bronchiolitis
Disease/ConditionBronchiolitis
Principal InvestigatorJacqueline Weingarten-Arams
Trial DescriptionEffect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial
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
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
Disease/ConditionAnaplastic Medulloblastoma
Medulloblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionEfficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients
Disease/ConditionAnaplastic Medulloblastoma
Medulloblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionEliminating Monitor Overuse (EMO) Hybrid Effectiveness-Deimplementation Trial
Disease/ConditionBronchiolitis Acute Viral
Principal InvestigatorAlyssa Silver
Trial DescriptionEstablishing Continuous Cell Lines and Xenografts From Pediatric Cancers for Biological and Pre-Clinical Therapeutic Studies
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 DescriptionEstablishing Continuous Cell Lines and Xenografts From Pediatric Cancers for Biological and Pre-Clinical Therapeutic Studies
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 DescriptionEvolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma
Disease/ConditionRhabdomyosarcoma
Principal InvestigatorAlice Lee
Trial DescriptionGenetic Modifiers of 22q11.2 Deletion Syndrome
Disease/ConditionDiGeorge Syndrome
22q11.2 Deletion Syndrome
Principal InvestigatorBernice Morrow
Trial DescriptionImplementing Models for Mechanical Circulatory Support Presurgical Assessment in Congenital Heart Disease Treatment
Disease/ConditionCongenital Heart Disease
Principal InvestigatorOmar Saeed
Trial DescriptionImproving Patient-Centered Communication in Breast Cancer: A RCT of a Shared Decision Engagement System (SHARES)
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
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
Disease/ConditionCentral Nervous System Neoplasm
Infantile Fibrosarcoma
Recurrent Acute Leukemia
Refractory Acute Leukemia
Solid Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionLarotrectinib (LOXO-101, NSC# 788607) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
Disease/ConditionCentral Nervous System Neoplasm
Infantile Fibrosarcoma
Recurrent Acute Leukemia
Refractory Acute Leukemia
Solid Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionLong-Term Follow-Up (LTFU) of Participants Treated With ADP Adoptive Cell Therapies
Disease/ConditionNeoplasms
Principal InvestigatorDavid Loeb
Trial DescriptionMetformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Principal Investigator
Trial DescriptionMinimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease
Disease/ConditionSickle Cell Disease
Principal InvestigatorMichelle Lee
Trial DescriptionMOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study
Disease/ConditionHemophilia A
Principal InvestigatorWilliam Mitchell
Trial DescriptionMolecular Analysis for Combination Therapy Choice (ComboMATCH)
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
Disease/ConditionHead and Neck Neoplasms
Principal InvestigatorRichard Smith
Trial DescriptionMolecular Basis of Early Childhood Obesity Programming by Intrauterine Growth Restriction
Disease/ConditionChildhood Obesity
Epigenetics
Principal InvestigatorMamta Fuloria
Trial DescriptionMulti-Center African-American Inflammatory Bowel Disease Study (MAAIS)
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
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
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, 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
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)
Disease/ConditionPrimary Immunodeficiency Diseases (PID)
Principal InvestigatorArye Rubinstein
Trial DescriptionMulti-Institution Registry of SRS/SBRT Procedures
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
Disease/ConditionHypoplastic Left Heart Syndrome (HLHS)
Principal InvestigatorNicole Sutton
Trial DescriptionNatural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections
Disease/ConditionMycobacterium Infections
Principal InvestigatorSteven Porcelli
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations
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
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
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
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
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
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
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
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
Disease/ConditionMinimal Change Disease (MCD)
Membranous Nephropathy
Glomerulosclerosis, Focal Segmental
Principal InvestigatorFrederick Kaskel
Trial DescriptionNeuroblastoma Biology Studies
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
Disease/ConditionChildhood Malignant Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionNMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)
Disease/ConditionNeuroblastoma
Principal InvestigatorDaniel Weiser
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry
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
Disease/ConditionH3 K27M
Glioma
Principal InvestigatorAdilia Hormigo
Trial DescriptionOptimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure?
Disease/ConditionPain, Procedural
Principal InvestigatorCourtney Mcnamara
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)
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 Peripheral Nerve Sheath Tumor
Malignant Skin Granular Cell 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
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 DescriptionPediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
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
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
Disease/ConditionSickle Cell Disease
Pain
Principal InvestigatorDeepa Manwani
Trial DescriptionPhase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis
Disease/ConditionRecurrent Langerhans Cell Histiocytosis
Refractory Langerhans Cell Histiocytosis
Principal InvestigatorAlice Lee
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
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 Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue
Disease/ConditionLocalized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Recurrent Neuroblastoma
Regional Neuroblastoma
Stage 4 Neuroblastoma
Stage 4S Neuroblastoma
Principal InvestigatorJonathan Gill
Trial DescriptionPhase III Randomized Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue
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
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
Disease/ConditionChronic Kidney Diseases
Principal InvestigatorFrederick Kaskel
Trial DescriptionProspective Cohort Study to Evaluate Immunologic Response Following COVID-19 Vaccination in Children, Adolescents and Young Adults With Cancer
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
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
Disease/ConditionCmv Congenital
CMV
Congenital Cmv
SNHL
Sensorineural Hearing Loss
Principal InvestigatorMichel Nassar
Trial DescriptionRandomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial
Disease/ConditionCmv Congenital
CMV
Congenital Cmv
SNHL
Sensorineural Hearing Loss
Principal InvestigatorMichel Nassar
Trial DescriptionRandomized Study of Intravenous Calaspargase Pegol (SC-PEG Asparaginase) and Intravenous Oncaspar in Children and Adolescents With Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma
Disease/ConditionAcute Lymphoblastic Leukemia
Lymphoblastic Lymphoma
Principal InvestigatorLisa Gennarini
Trial DescriptionREal-World Analyses of Stroke - Thrombus Occlusion REtrieval
Disease/ConditionAcute Ischemic Stroke
Vessel Occlusion
Stroke
Ischemic
Principal InvestigatorNeil Haranhalli
Trial DescriptionRegistry of Asthma Patients Initiating DUPIXENT® (RAPID)
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionRegistry of Asthma Patients Initiating DUPIXENT® (RAPID)
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.
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-Stratified Randomized Phase III Testing of Blinatumomab (NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionSafety and Effectiveness of Balloon-Expandable Bioprosthetic SAPIEN X4 Transcatheter Heart Valve
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
Disease/ConditionSickle Cell Disease
Principal InvestigatorDeepa Manwani
Trial DescriptionSimple Bone Cysts in Kids (SBoCK)
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
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)
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
Disease/ConditionTransplant Rejection
Allograft Rejection
Complications
Principal InvestigatorNicole Hayde
Trial DescriptionSurveillance HeartCare® Outcomes Registry
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionSurveillance HeartCare® Outcomes Registry
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionTesting the Feasibility of a Teletherapy Plus Parent Coaching Intervention for Children With Autism
Disease/ConditionAutism Spectrum Disorder
Principal InvestigatorElizabeth Ridgway
Trial DescriptionThe Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study
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
Disease/ConditionScoliosis Idiopathic
Principal InvestigatorJaime Gomez
Trial DescriptionTreatment for Patients With Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor
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 for Patients With Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor
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
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
Disease/ConditionAcute Lymphoblastic Leukemia, Pediatric
Principal InvestigatorLisa Gennarini
Trial DescriptionTreatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
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 DescriptionTrialNet Pathway to Prevention of T1D
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
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionTumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial
Disease/ConditionSolid Tumors
Principal InvestigatorHaiying Cheng
Trial DescriptionUmbrella Long-Term Follow-Up Protocol
Disease/ConditionHematopoietic Cell Transplantation Recipient
Leukemia
Solid Tumor
Principal InvestigatorAlice Lee
Trial DescriptionUS Selumetinib Registry
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
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)
Disease/ConditionCoronavirus
Principal InvestigatorJen-Ting Chen