Trial DescriptionA 52-week, Randomised, Double-blind, Double-dummy, Parallel Group, Multi-centre, Non-inferiority Study Assessing Exacerbation Rate, Additional Measures of Asthma Control and Safety in Adult and Adolescent Severe Asthmatic Participants With an Eosinophilic Phenotype Treated With GSK3511294 Compared With Mepolizumab or Benralizumab - This is randomized, double-blind, parallel group, multi-center and non-inferiority study.NCT04718389
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 52-week, Randomised, Double-blind, Placebo-controlled, Parallel-group, Multi-centre Study of the Efficacy and Safety of GSK3511294 Adjunctive Therapy in Adult and Adolescent Participants With Severe Uncontrolled Asthma With an Eosinophilic Phenotype - Participants will be randomized in a 2:1 ratio to receive either GSK3511294 (Depemokimab) or placebo as an adjunct therapy.NCT04718103
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 52-week, Randomised, Double-blind, Placebo-controlled, Parallel-group, Multi-centre Study of the Efficacy and Safety of GSK3511294 Adjunctive Therapy in Adult and Adolescent Participants With Severe Uncontrolled Asthma With an Eosinophilic Phenotype - Participants will be randomized in a 2:1 ratio to receive either GSK3511294 (Depemokimab) or placebo as an adjunct therapy.NCT04718103
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionA 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 Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy NCT04336722
Disease/ConditionBiliary Atresia
Principal InvestigatorDebra Pan
Trial DescriptionA Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy NCT04336722
Disease/ConditionBiliary Atresia
Principal InvestigatorDebra Pan
Trial DescriptionA Longitudinal Observational Study of Patients Undergoing Therapy for Immune-Mediated Inflammatory Skin Conditions NCT03661866
Disease/ConditionAtopic Dermatitis
Alopecia Areata
Hidradenitis Suppurativa
Vitiligo
Psoriasis
Chronic Spontaneous Urticaria
Principal InvestigatorHolly Kanavy
Trial DescriptionA Longitudinal Observational Study of Patients 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 Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution NCT05044819
Disease/ConditionLennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Principal InvestigatorPuja Patel
Trial DescriptionA 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 Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications NCT01351545
Disease/ConditionHematologic Malignancies
Inherited Disorders of Metabolism
Inherited Abnormalities of Platelets
Histiocytic Disorders
Acute Myelogenous Leukemia (AML or ANLL)
Acute Lymphoblastic Leukemia (ALL)
Other Acute Leukemia
Chronic Myelogenous Leukemia (CML)
Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases
Other Leukemia
Hodgkin Lymphoma
Non-hodgkin Lymphoma
Multiple Myeloma/ Plasma Cell Disorder (PCD)
Inherited Abnormalities of Erythrocyte Differentiation or Function
Disorders of the Immune System
Autoimmune Diseases
Severe Aplastic Anemia
Principal InvestigatorDavid Loeb
Trial DescriptionA Multicenter Pilot Study of Reduced Intensity Conditioning and Allogeneic Stem Cell Transplantation Followed by Targeted Immunotherapy in Children, Adolescents and Young Adults With Poor Risk CD30+ Hodgkin Lymphoma (HL) NCT02098512
Disease/ConditionHodgkin Lymphoma
Principal InvestigatorKris Mahadeo
Trial DescriptionA Multicenter Pilot Study of Reduced Intensity Conditioning and Allogeneic Stem Cell Transplantation Followed by Targeted Immunotherapy in Children, Adolescents and Young Adults With Poor Risk CD30+ Hodgkin Lymphoma (HL) NCT02098512
Disease/ConditionHodgkin Lymphoma
Principal InvestigatorKris Mahadeo
Trial DescriptionA Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic Syndrome (MDS) NCT05457556
Disease/ConditionAcute Lymphoblastic Leukemia
Acute Myeloid Leukemia
Mixed Phenotype Acute Leukemia
Myelodysplastic Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionA Multicenter, Prospective, Long-term, Observational Registry of Pediatric Patients With Inflammatory Bowel Disease NCT00606346
Disease/ConditionCrohn's Disease
Ulcerative Colitis
Indeterminate Colitis
Inflammatory Bowel Diseases
Principal InvestigatorGitit Tomer
Trial DescriptionA Multi-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-Institution Study of TGFβ Imprinted, Ex Vivo Expanded Universal Donor NK Cell Infusions as Adoptive Immunotherapy in Combination With Gemcitabine and Docetaxel in Patients With Relapsed or Refractory Pediatric Bone and Soft Tissue - Single arm, unblinded, phase I/II studyNCT05634369
Disease/ConditionPediatric Sarcoma, Refractory
Pediatric Sarcoma, Relapsed
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 1, First-in-Human, Dose Escalation and Expansion Study to Evaluate the Safety and Tolerability of XmAb®541 in Advanced Solid Tumors - Dose escalation and expansion studyNCT06276491
Disease/ConditionOvarian Cancer
Endometrial Cancer
Germ Cell Tumor
Testicular Germ Cell Tumor
Ovarian Germ Cell Tumor
Principal InvestigatorEric Feldman
Trial DescriptionA Phase 1, First-in-Human, Dose Escalation and Expansion Study to Evaluate the Safety and Tolerability of XmAb®541 in Advanced Solid Tumors - Dose escalation and expansion studyNCT06276491
Disease/ConditionOvarian Cancer
Endometrial Cancer
Germ Cell Tumor
Testicular Germ Cell Tumor
Ovarian Germ Cell Tumor
Principal InvestigatorEric Feldman
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, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients With Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation - Phase 1 will employ an accelerated titration design. The dose escalation will follow a modified Fibonacci sequence.NCT04065399
Disease/ConditionAcute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Mixed Lineage Acute Leukemia
Mixed Phenotype Acute Leukemia
Acute Leukemia of Ambiguous Lineage
Principal InvestigatorIoannis Mantzaris
Trial DescriptionA Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients With Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation - Phase 1 will employ an accelerated titration design. The dose escalation will follow a modified Fibonacci sequence.NCT04065399
Disease/ConditionAcute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Mixed Lineage Acute Leukemia
Mixed Phenotype Acute Leukemia
Acute Leukemia of Ambiguous Lineage
Principal InvestigatorIoannis Mantzaris
Trial DescriptionA Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma NCT03794349
Disease/ConditionHigh Risk Neuroblastoma
Recurrent Neuroblastoma
Refractory Neuroblastoma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG) NCT03919071
Disease/ConditionAnaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Anaplastic Ganglioglioma
Anaplastic Pleomorphic Xanthoastrocytoma
Glioblastoma
Malignant Glioma
WHO Grade 3 Glioma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG) NCT03919071
Disease/ConditionAnaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Anaplastic Ganglioglioma
Anaplastic Pleomorphic Xanthoastrocytoma
Glioblastoma
Malignant Glioma
WHO Grade 3 Glioma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518) in Children and Young Adults With Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL) NCT02981628
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Recurrent B Lymphoblastic Lymphoma
Refractory B Acute Lymphoblastic Leukemia
Refractory B Lymphoblastic Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 2 Study of 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,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 Randomized, Placebo-Controlled, Double-blind, Clinical Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Systemic Left Ventricular Systolic Dysfunction (VALOR) NCT05714085
Disease/ConditionHeart Failure
Left Ventricular Systolic Dysfunction
Principal InvestigatorDaphne Hsu
Trial DescriptionA Phase 2/3, Open-Label Study of the Pharmacokinetics, Safety, and Antiviral Activity of the Elvitegravir/Cobicistat/Emtricitabine/Tenofovir Alafenamide (E/C/F/TAF) Single Tablet Regimen (STR) in HIV-1 Infected Antiretroviral Treatment-Naive Adolescents and Virologically Suppressed Children NCT01854775
Disease/ConditionAcquired Immune Deficiency Syndrome (AIDS)
HIV Infections
Principal InvestigatorDonna Futterman
Trial DescriptionA Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1) NCT04166409
Disease/ConditionLow Grade Astrocytoma
Low Grade Glioma
Metastatic Low Grade Astrocytoma
Metastatic Low Grade Glioma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) NCT03871257
Disease/ConditionLow Grade Glioma
Neurofibromatosis Type 1
Visual Pathway Glioma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations NCT04293562
Disease/ConditionAcute Myeloid Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations NCT04293562
Disease/ConditionAcute Myeloid Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Randomized Trial of Inotuzumab Ozogamicin (NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy NCT03959085
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Central Nervous System Leukemia
Mixed Phenotype Acute Leukemia
Testicular Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Randomized Trial of Inotuzumab Ozogamicin (NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy NCT03959085
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Central Nervous System Leukemia
Mixed Phenotype Acute Leukemia
Testicular Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Upadacitinib in Adolescent and Adult Subjects With Moderate to Severe Atopic Dermatitis NCT03569293
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
Trial DescriptionA Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients With Germ Cell Tumors NCT03067181
Disease/ConditionChildhood Extracranial Germ Cell Tumor
Extragonadal Embryonal Carcinoma
Germ Cell Tumor
Malignant Germ Cell Tumor
Malignant Ovarian Teratoma
Stage I Ovarian Choriocarcinoma
Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage I Ovarian Teratoma AJCC v6 and v7
Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage I Testicular Choriocarcinoma AJCC v6 and v7
Stage I Testicular Embryonal Carcinoma AJCC v6 and v7
Stage I Testicular Seminoma AJCC v6 and v7
Stage I Testicular Yolk Sac Tumor AJCC v6 and v7
Stage II Ovarian Choriocarcinoma
Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage II Testicular Choriocarcinoma AJCC v6 and v7
Stage II Testicular Embryonal Carcinoma AJCC v6 and v7
Stage II Testicular Yolk Sac Tumor AJCC v6 and v7
Stage III Ovarian Choriocarcinoma
Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7
Stage III Testicular Choriocarcinoma AJCC v6 and v7
Stage III Testicular Embryonal Carcinoma AJCC v6 and v7
Stage III Testicular Yolk Sac Tumor AJCC v6 and v7
Stage IV Ovarian Choriocarcinoma
Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7
Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7
Testicular Mixed Choriocarcinoma and Embryonal Carcinoma
Testicular Mixed Choriocarcinoma and Teratoma
Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3 Trial Investigating Blinatumomab (NSC# 765986) in Combination With Chemotherapy in Patients With Newly Diagnosed Standard Risk or Down Syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients With Localized B-Lymphoblastic Lymphoma (B-LLy) NCT03914625
Disease/ConditionB Acute Lymphoblastic Leukemia
B Lymphoblastic Lymphoma
Down Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionA Phase 3, Double-Blind, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Subcutaneous Twice Yearly Long-Acting Lenacapavir for HIV Pre-Exposure Prophylaxis in Cisgender Men, Transgender Women, Transgender Men, and Gender Nonbinary People ≥ 16 Years of Age Who Have Sex With Male Partners and Are at Risk for HIV Infection NCT04925752
Disease/ConditionPre-Exposure Prophylaxis of HIV Infection
Principal InvestigatorMichelle Collins-Ogle
Trial DescriptionA Phase 3, Double-Blind, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Subcutaneous Twice Yearly Long-Acting Lenacapavir for HIV Pre-Exposure Prophylaxis in Cisgender Men, Transgender Women, Transgender Men, and Gender Nonbinary People ≥ 16 Years of Age Who Have Sex With Male Partners and Are at Risk for HIV Infection NCT04925752
Disease/ConditionPre-Exposure Prophylaxis of HIV Infection
Principal InvestigatorMichelle Collins-Ogle
Trial DescriptionA Phase 3, Double-Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics (PK) of Baricitinib in Children From 6 Years to Less Than 18 Years of Age With Alopecia Areata NCT05723198
Disease/ConditionAreata Alopecia
Alopecia
Hypotrichosis
Hair Diseases
Skin Diseases
Pathological Conditions, Anatomical
Principal InvestigatorHolly Kanavy
Trial DescriptionA Phase 3, Double-Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics (PK) of Baricitinib in Children From 6 Years to Less Than 18 Years of Age With Alopecia Areata NCT05723198
Disease/ConditionAreata Alopecia
Alopecia
Hypotrichosis
Hair Diseases
Skin Diseases
Pathological Conditions, Anatomical
Principal InvestigatorHolly Kanavy
Trial DescriptionA Phase 3, Multicenter, Open-Label, Single Arm Study of MR-100A-01 in Women of Childbearing Potential to Evaluate Contraceptive Efficacy and Safety NCT05139121
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
Trial DescriptionA Phase 3, Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel-Group Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) NCT04562766
Disease/ConditionImmune Thrombocytopenia
Principal InvestigatorIrina Murakhovskaya
Trial DescriptionA Phase 3, Open-label, Multi-center, Single Arm Study to Assess Contraceptive Efficacy and Safety of the Etonogestrel (MK-8415) Implant During Extended Use From 3 Years After Insertion in Females 35 Years of Age or Younger NCT04626596
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
Trial DescriptionA Phase 3, Open-label, Multi-center, Single Arm Study to Assess Contraceptive Efficacy and Safety of the Etonogestrel (MK-8415) Implant During Extended Use From 3 Years After Insertion in Females 35 Years of Age or Younger NCT04626596
Disease/ConditionContraception
Principal InvestigatorJessica Atrio
Trial DescriptionA Phase 3, Open-label, Randomized, Multicenter, 12 Months, Efficacy and Safety Study of Weekly MOD-4023 Compared to Daily Genotropin - Therapy in Pre-pubertal Children With Growth Hormone Deficiency NCT02968004
Disease/ConditionPediatric Growth Hormone Deficiency
Principal InvestigatorPing Zhou
Trial DescriptionA Phase 3, Randomized, 52-week, Placebo-controlled, Double-blind Study With Rerandomization to Assess the Efficacy, Safety, and Tolerability of Rocatinlimab (AMG 451) in Adolescent Subjects With Moderate-to-severe Atopic Dermatitis (AD) (ROCKET-ASTRO) NCT05704738
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
Trial DescriptionA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib Administered Once Daily for 52 Weeks in Subjects With Non-Cystic Fibrosis Bronchiectasis - The ASPEN Study NCT04594369
Disease/ConditionNon-Cystic Fibrosis Bronchiectasis
Principal InvestigatorDivya Reddy
Trial DescriptionA Phase 3b, Multi Center, Open-label, Long-term Extension Study of Apremilast (CC-10004) in Pediatric Subjects From 6 Through 17 Years of Age With Moderate to Severe Plaque Psoriasis NCT04175613
Disease/ConditionPsoriasis
Principal InvestigatorJulia Gittler
Trial DescriptionA Phase 3b/4 Randomized, Open-label, Efficacy Assessor Blinded Study, Comparing the Safety and Assessor Blinded Efficacy of Upadacitinib to Dupilumab in Subjects With Moderate to Severe Atopic Dermatitis (Level-Up) NCT05601882
Disease/ConditionAtopic Dermatitis
Principal InvestigatorHolly Kanavy
Trial DescriptionA Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination With Azacitidine in Patients With Recurrent, Resectable Osteosarcoma - Phase Ib lead-in with extension to Phase IINCT03628209
Disease/ConditionOsteosarcoma
Osteosarcoma in Children
Osteosarcoma Recurrent
Sarcoma
Principal InvestigatorDaniel Weiser
Trial DescriptionA Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination With Azacitidine in Patients With Recurrent, Resectable Osteosarcoma - Phase Ib lead-in with extension to Phase IINCT03628209
Disease/ConditionOsteosarcoma
Osteosarcoma in Children
Osteosarcoma Recurrent
Sarcoma
Principal InvestigatorDaniel Weiser
Trial DescriptionA Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of RO7017773 in Participants Aged 15-45 Years With Autism Spectrum Disorder (ASD) NCT04299464
Disease/ConditionAutism Spectrum Disorder (ASD)
Principal InvestigatorEric Hollander
Trial DescriptionA Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of RO7017773 in Participants Aged 15-45 Years With Autism Spectrum Disorder (ASD) NCT04299464
Disease/ConditionAutism Spectrum Disorder (ASD)
Principal InvestigatorEric Hollander
Trial DescriptionA Phase 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 Trial for Newly Diagnosed High Risk B-Lymphoblastic Leukemia (B-ALL) Including a Stratum Evaluating Dasatinib (NSC#732517) in Patients With Ph-like Tyrosine Kinase Inhibitor (TKI) Sensitive Mutations NCT02883049
Disease/ConditionB Acute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia, BCR-ABL1-Like
Central Nervous System Leukemia
Testicular Leukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial for Newly Diagnosed High Risk B-Lymphoblastic Leukemia (B-ALL) Including a Stratum Evaluating Dasatinib (NSC#732517) in Patients With Ph-like Tyrosine Kinase Inhibitor (TKI) Sensitive Mutations NCT02883049
Disease/ConditionB Acute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia, BCR-ABL1-Like
Central Nervous System Leukemia
Testicular Leukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial for Patients With De Novo AML Using Bortezomib and Sorafenib (NSC# 681239, NSC# 724772) for Patients With High Allelic Ratio FLT3/ITD NCT01371981
Disease/ConditionAcute Myeloid Leukemia
Leukemia Cutis
Myeloid Neoplasm
Myeloid Sarcoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial for Patients With De Novo AML Using Bortezomib and Sorafenib (NSC# 681239, NSC# 724772) for Patients With High Allelic Ratio FLT3/ITD NCT01371981
Disease/ConditionAcute Myeloid Leukemia
Leukemia Cutis
Myeloid Neoplasm
Myeloid Sarcoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial 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 Randomized Trial Investigating Bortezomib (NSC# 681239) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy) NCT02112916
Disease/ConditionAdult T Acute Lymphoblastic Leukemia
Ann Arbor Stage II Adult Lymphoblastic Lymphoma
Ann Arbor Stage II Childhood Lymphoblastic Lymphoma
Ann Arbor Stage III Adult Lymphoblastic Lymphoma
Ann Arbor Stage III Childhood Lymphoblastic Lymphoma
Ann Arbor Stage IV Adult Lymphoblastic Lymphoma
Ann Arbor Stage IV Childhood Lymphoblastic Lymphoma
Childhood T Acute Lymphoblastic Leukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial Investigating Bortezomib (NSC# 681239) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy) NCT02112916
Disease/ConditionAdult T Acute Lymphoblastic Leukemia
Ann Arbor Stage II Adult Lymphoblastic Lymphoma
Ann Arbor Stage II Childhood Lymphoblastic Lymphoma
Ann Arbor Stage III Adult Lymphoblastic Lymphoma
Ann Arbor Stage III Childhood Lymphoblastic Lymphoma
Ann Arbor Stage IV Adult Lymphoblastic Lymphoma
Ann Arbor Stage IV Childhood Lymphoblastic Lymphoma
Childhood T Acute Lymphoblastic Leukemia
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial of Adding Vincristine-Topotecan-Cyclophosphamide to Standard Chemotherapy in Initial Treatment of Non-Metastatic Ewing Sarcoma NCT01231906
Disease/ConditionLocalized Extraskeletal Ewing Sarcoma
Peripheral Primitive Neuroectodermal Tumor of Bone
Peripheral Primitive Neuroectodermal Tumor of Soft Tissues
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III Randomized Trial of Adding Vincristine-Topotecan-Cyclophosphamide to Standard Chemotherapy in Initial Treatment of Non-Metastatic Ewing Sarcoma NCT01231906
Disease/ConditionLocalized Extraskeletal Ewing Sarcoma
Peripheral Primitive Neuroectodermal Tumor of Bone
Peripheral Primitive Neuroectodermal Tumor of Soft Tissues
Principal InvestigatorLisa Gennarini
Trial DescriptionA Phase III, Randomized Study of Nivolumab (Opdivo) Plus AVD or Brentuximab Vedotin (Adcetris) Plus AVD in Patients (Age >/= 12 Years) With Newly Diagnosed Advanced Stage Classical Hodgkin Lymphoma NCT03907488
Disease/ConditionAnn Arbor Stage III Hodgkin Lymphoma
Ann Arbor Stage III Lymphocyte-Depleted Classic Hodgkin Lymphoma
Ann Arbor Stage III Mixed Cellularity Classic Hodgkin Lymphoma
Ann Arbor Stage III Nodular Sclerosis Classic Hodgkin Lymphoma
Ann Arbor Stage IV Hodgkin Lymphoma
Ann Arbor Stage IV Lymphocyte-Depleted Classic Hodgkin Lymphoma
Ann Arbor Stage IV Mixed Cellularity Classic Hodgkin Lymphoma
Ann Arbor Stage IV Nodular Sclerosis Classic Hodgkin Lymphoma
Classic Hodgkin Lymphoma
Lymphocyte-Rich Classic Hodgkin Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma NCT04385277
Disease/ConditionGanglioneuroblastoma, Nodular
Neuroblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA 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, Multi-center Registry for Patients With Short Bowel Syndrome NCT01990040
Disease/ConditionShort Bowel Syndrome
Principal InvestigatorJohn Thompson
Trial DescriptionA Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid) NCT03115086
Disease/ConditionBile Acid Synthesis Disorders
Principal InvestigatorNadia Ovchinsky
Trial DescriptionA Prospective, Open-label, Randomized, Concurrent Active-controlled, Longitudinal, Multicenter, Phase 3 Clinical Study of the Safety and Efficacy of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee NCT03588975
Disease/ConditionChondral Defect
Osteochondritis Dissecans (OCD)
Articular Cartilage Defect
Articular Cartilage Disorder of Knee
Principal InvestigatorEric Fornari
Trial DescriptionA Randomized Phase 2 Trial of Brentuximab Vedotin (SGN35, NSC# 749710), or Crizotinib (NSC#749005, Commercially Labeled) in Combination With Chemotherapy for Newly Diagnosed Patients With Anaplastic Large Cell Lymphoma (ALCL) NCT01979536
Disease/ConditionAnaplastic Large Cell Lymphoma, ALK-Positive
Ann Arbor Stage II Noncutaneous Childhood Anaplastic Large Cell Lymphoma
Ann Arbor Stage III Noncutaneous Childhood Anaplastic Large Cell Lymphoma
Ann Arbor Stage IV Noncutaneous Childhood Anaplastic Large Cell Lymphoma
Principal InvestigatorLisa Gennarini
Trial DescriptionA Randomized Phase 3 Interim Response Adapted Trial Comparing Standard Therapy With Immuno-oncology Therapy for Children and Adults With Newly Diagnosed Stage I and II Classic Hodgkin Lymphoma NCT05675410
Disease/ConditionLugano Classification Limited Stage Hodgkin Lymphoma AJCC v8
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized Phase 3 Study of Brentuximab Vedotin (SGN-35) for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma (cHL) in Children and Young Adults NCT02166463
Disease/ConditionAnn Arbor Stage IIB Hodgkin Lymphoma
Ann Arbor Stage IIIB Hodgkin Lymphoma
Ann Arbor Stage IVA Hodgkin Lymphoma
Ann Arbor Stage IVB Hodgkin Lymphoma
Childhood Hodgkin Lymphoma
Classic Hodgkin Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized Phase 3 Study of Brentuximab Vedotin (SGN-35) for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma (cHL) in Children and Young Adults NCT02166463
Disease/ConditionAnn Arbor Stage IIB Hodgkin Lymphoma
Ann Arbor Stage IIIB Hodgkin Lymphoma
Ann Arbor Stage IVA Hodgkin Lymphoma
Ann Arbor Stage IVB Hodgkin Lymphoma
Childhood Hodgkin Lymphoma
Classic Hodgkin Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS) NCT02567435
Disease/ConditionAlveolar Rhabdomyosarcoma
Botryoid-Type Embryonal Rhabdomyosarcoma
Embryonal Rhabdomyosarcoma
Rhabdomyosarcoma
Sclerosing Rhabdomyosarcoma
Spindle Cell Rhabdomyosarcoma
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized Phase 3 Trial of Nivolumab (NSC# 748726) in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma NCT04759586
Disease/ConditionPrimary Mediastinal Large B-Cell Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized Phase III Trial Comparing Conventional-Dose Chemotherapy Using Paclitaxel, Ifosfamide, and Cisplatin (TIP) With High-Dose Chemotherapy Using Mobilizing Paclitaxel Plus Ifosfamide Followed by High-Dose Carboplatin and Etoposide (TI-CE) as First Salvage Treatment in Relapsed or Refractory Germ Cell Tumors NCT02375204
Disease/ConditionGerm Cell Tumor
Teratoma
Choriocarcinoma
Germinoma
Mixed Germ Cell Tumor
Yolk Sac Tumor
Childhood Teratoma
Malignant Germ Cell Neoplasm
Extragonadal Seminoma
Non-seminomatous Germ Cell Tumor
Seminoma
Principal InvestigatorBenjamin Gartrell
Trial DescriptionA Randomized Trial of Levocarnitine Prophylaxis to Prevent Asparaginase-Associated Hepatotoxicity in Adolescents and Young Adults Receiving Acute Lymphoblastic Leukemia Therapy NCT05602194
Disease/ConditionB Acute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1
B Acute Lymphoblastic Leukemia, BCR-ABL1-Like
Lymphoblastic Lymphoma
Mixed Phenotype Acute Leukemia
T Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionA Randomized, Assessor-Blind, Parallel-Groups, Multicenter Trial Assessing the Safety and Efficacy, Including Pharmacokinetic Assessments, of CLENPIQ in Children Aged 2 Years to Less Than 9 Years NCT04113382
Disease/ConditionBowel Preparation
Principal InvestigatorInna Novak
Trial DescriptionA Randomized, 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 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, Multicenter, Double-blind, Parallel, Active-control Study of the Effects of Sparsentan, a Dual Endothelin Receptor and Angiotensin Receptor Blocker, on Renal Outcomes in Patients With Primary FSGS NCT03493685
Disease/ConditionFocal Segmental Glomerulosclerosis
Principal InvestigatorFrederick Kaskel
Trial DescriptionA Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in Combination With Azacitidine After Allogeneic Stem Cell Transplantation in Subjects With Acute Myeloid Leukemia (AML) (VIALE-T) NCT04161885
Disease/ConditionAcute Myeloid Leukemia (AML)
Cancer
Principal InvestigatorIoannis Mantzaris
Trial DescriptionA Remotely Delivered CBT Intervention for Youth With cSLE: A Multi-site Patient-engaged Investigation - Participants will either be randomly assigned to the intervention or to a wait-list period.NCT04335643
Disease/ConditionSystemic Lupus Erythematosus
Principal InvestigatorTamar Rubinstein
Trial DescriptionA Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors NCT04195399
Disease/ConditionDesmoid Fibromatosis
Recurrent Desmoid Fibromatosis
Unresectable Desmoid Fibromatosis
Principal InvestigatorLisa Gennarini
Trial DescriptionA 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 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, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA) NCT03841357
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
Trial DescriptionAn Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA) NCT03841357
Disease/ConditionJuvenile Idiopathic Arthritis
Principal InvestigatorDawn Wahezi
Trial DescriptionAn Open-Label 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 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, 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 DescriptionAssessment of CMV-specific T Cell Responses by Flow Cytometry With Intracellular Cytokine Staining to Predict CMV Infection Risk in Pediatric Solid Organ Transplant Recipients NCT03924219
Disease/ConditionHeart Transplant Infection
Kidney Transplant Infection
Liver Transplant Infection
CMV
Principal InvestigatorBetsy Herold
Trial DescriptionChronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors) NCT05702645
Disease/ConditionB Acute Lymphoblastic Leukemia Associated With Down Syndrome
Down Syndrome
Myeloid Leukemia Associated With Down Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionChronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors) NCT05702645
Disease/ConditionB Acute Lymphoblastic Leukemia Associated With Down Syndrome
Down Syndrome
Myeloid Leukemia Associated With Down Syndrome
Principal InvestigatorAlice Lee
Trial DescriptionComparison of Conventional Non-Invasive Ventilation and Neurally-Adjusted Ventilatory Assistance (NAVA) Non-Invasive Ventilation for the Treatment of Bronchiolitis - Non-blinded, randomized clinical trialNCT06053684
Disease/ConditionBronchiolitis
Principal InvestigatorJacqueline Weingarten-Arams
Trial DescriptionComparison of Conventional Non-Invasive Ventilation and Neurally-Adjusted Ventilatory Assistance (NAVA) Non-Invasive Ventilation for the Treatment of Bronchiolitis - Non-blinded, randomized clinical trialNCT06053684
Disease/ConditionBronchiolitis
Principal InvestigatorJacqueline Weingarten-Arams
Trial DescriptionCOVID-19 Vaccine Response in People Living With Sickle Cell Disease NCT05139992
Disease/ConditionSickle Cell Disease
COVID-19
Principal InvestigatorDeepa Manwani
Trial DescriptionEffect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial - This is a genotype stratified, double-blind, randomized, placebo-controlled, parallel-group, phase IV clinical trialNCT03694158
Disease/ConditionAsthma
Principal InvestigatorSunit Jariwala
Trial DescriptionEffect of IL-4RαR576 Polymorphism on Response to Dupilumab in Asthma, a Genotype-stratified, Randomized, Placebo- Controlled Trial - This is a genotype stratified, double-blind, randomized, placebo-controlled, parallel-group, phase IV clinical trialNCT03694158
Disease/ConditionAsthma
Principal InvestigatorSunit Jariwala
Trial 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 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 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 DescriptionEvolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma NCT04388839
Disease/ConditionRhabdomyosarcoma
Principal InvestigatorAlice Lee
Trial DescriptionGenetic Modifiers of 22q11.2 Deletion Syndrome NCT00556530
Disease/ConditionDiGeorge Syndrome
22q11.2 Deletion Syndrome
Principal InvestigatorBernice Morrow
Trial DescriptionInternational Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones NCT03007147
Disease/ConditionAcute Lymphoblastic Leukemia
B Acute Lymphoblastic Leukemia
Mixed Phenotype Acute Leukemia
T Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionJ-Valve TF Early Feasibility Study NCT06034028
Disease/ConditionAortic Valve Disease
Aortic Valve Insufficiency
Aortic Regurgitation
Principal InvestigatorMohamed Azeem Latib
Trial DescriptionJ-Valve TF Early Feasibility Study NCT06034028
Disease/ConditionAortic Valve Disease
Aortic Valve Insufficiency
Aortic Regurgitation
Principal InvestigatorMohamed Azeem Latib
Trial DescriptionLarotrectinib (LOXO-101, NSC# 788607) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias NCT03834961
Disease/ConditionCentral Nervous System Neoplasm
Infantile Fibrosarcoma
Recurrent Acute Leukemia
Refractory Acute Leukemia
Solid Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionLongitudinal Assessment of Ovarian Reserve in Adolescents With Lymphoma NCT01793233
Disease/ConditionAnaplastic Large Cell Lymphoma
Burkitt Lymphoma
Diffuse Large B-Cell Lymphoma
Hodgkin Lymphoma
Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionLongitudinal Assessment of Ovarian Reserve in Adolescents With Lymphoma NCT01793233
Disease/ConditionAnaplastic Large Cell Lymphoma
Burkitt Lymphoma
Diffuse Large B-Cell Lymphoma
Hodgkin Lymphoma
Lymphoma
Principal InvestigatorAlice Lee
Trial DescriptionMatched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome NCT02670525
Disease/ConditionRecurrent, Refractory, or High Risk Leukemias
Matched Targeted Therapy
Principal InvestigatorLisa Gennarini
Trial DescriptionMetformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse - Group 1 Localized osteosarcoma that have presented a response ≤ 60% to the pre-operatory chemotherapy.
Is expected the enrolment of 37 patients as total population, in 44 months. The patients will be followed for 3 years, started from the date of enrolment. Metformin will be administrated for 3 years maximum, unless there is a progression disease or if toxicity is verified.
Group 2 Osteosarcoma and Ewing sarcoma patients with complete remission after the first relapse.
Is expected the enrolment of 30 patients as total population, in 24 months. Metformin will be administrated at the Group 2 for 1 year. If there is no toxicity verified or relapse, the patient should continue the treatment for other 2 year (in a total 3 years as maximum).NCT04758000
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Principal Investigator
Trial DescriptionMetformin as Maintenance Therapy in Patients With Bone Sarcoma and High Risk of Relapse - Group 1 Localized osteosarcoma that have presented a response ≤ 60% to the pre-operatory chemotherapy.
Is expected the enrolment of 37 patients as total population, in 44 months. The patients will be followed for 3 years, started from the date of enrolment. Metformin will be administrated for 3 years maximum, unless there is a progression disease or if toxicity is verified.
Group 2 Osteosarcoma and Ewing sarcoma patients with complete remission after the first relapse.
Is expected the enrolment of 30 patients as total population, in 24 months. Metformin will be administrated at the Group 2 for 1 year. If there is no toxicity verified or relapse, the patient should continue the treatment for other 2 year (in a total 3 years as maximum).NCT04758000
Disease/ConditionOsteosarcoma
Ewing Sarcoma
Principal Investigator
Trial DescriptionMinimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease NCT03587272
Disease/ConditionSickle Cell Disease
Principal InvestigatorMichelle Lee
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 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, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Subjects With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure of Rituximab or Rituximab and Chemotherapy NCT03394365
Disease/ConditionEpstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
Solid Organ Transplant Complications
Lymphoproliferative Disorders
Allogeneic Hematopoietic Cell Transplant
Stem Cell Transplant Complications
Principal InvestigatorIoannis Mantzaris
Trial DescriptionMulticenter, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Subjects With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure of Rituximab or Rituximab and Chemotherapy NCT03394365
Disease/ConditionEpstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
Solid Organ Transplant Complications
Lymphoproliferative Disorders
Allogeneic Hematopoietic Cell Transplant
Stem Cell Transplant Complications
Principal InvestigatorIoannis Mantzaris
Trial DescriptionMulticenter, Prospective, Open-label, Randomized, Crossover Study to Evaluate Pharmacokinetics (PK), Safety, and Tolerability of TAK-881 in Primary Immunodeficiency Diseases (PIDD) - This study consists of crossover randomized epoch (participants aged >=16 years) and single arm epoch (participants aged 2 to <16 years).NCT05755035
Disease/ConditionPrimary Immunodeficiency Diseases (PID)
Principal InvestigatorArye Rubinstein
Trial DescriptionMulticenter, Prospective, Open-label, Randomized, Crossover Study to Evaluate Pharmacokinetics (PK), Safety, and Tolerability of TAK-881 in Primary Immunodeficiency Diseases (PIDD) - This study consists of crossover randomized epoch (participants aged >=16 years) and single arm epoch (participants aged 2 to <16 years).NCT05755035
Disease/ConditionPrimary Immunodeficiency Diseases (PID)
Principal InvestigatorArye Rubinstein
Trial DescriptionMulti-Institution Registry of SRS/SBRT Procedures NCT01885299
Disease/ConditionNeoplasms
Arteriovenous Malformation of Central Nervous System
Trigeminal Neuralgia
Principal InvestigatorMadhur Garg
Trial DescriptionMulti-Institution Registry of SRS/SBRT Procedures NCT01885299
Disease/ConditionNeoplasms
Arteriovenous Malformation of Central Nervous System
Trigeminal Neuralgia
Principal InvestigatorMadhur Garg
Trial DescriptionNational Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children With Complex Congenital Heart Disease NCT02852031
Disease/ConditionHypoplastic Left Heart Syndrome (HLHS)
Principal InvestigatorNicole Sutton
Trial DescriptionNatural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections NCT00018044
Disease/ConditionMycobacterium Infections
Principal InvestigatorSteven Porcelli
Trial DescriptionNatural History, Genetics, Phenotype, and Treatment of Mycobacterial Infections NCT00018044
Disease/ConditionMycobacterium Infections
Principal InvestigatorSteven Porcelli
Trial DescriptionNCI COVID-19 in Cancer Patients Study (NCCAPS): A Longitudinal Natural History Study NCT04387656
Disease/ConditionCOVID-19 Infection
Hematopoietic and Lymphatic System Neoplasm
Malignant Solid Neoplasm
Metastatic Malignant Solid Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations NCT04195555
Disease/ConditionRecurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Rhabdoid Tumor
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Recurrent WHO Grade 2 Glioma
Refractory Ependymoma
Refractory Ewing Sarcoma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Refractory WHO Grade 2 Glioma
Wilms Tumor
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Erdafitinib in Patients With Tumors Harboring FGFR1/2/3/4 Alterations NCT03210714
Disease/ConditionAdvanced Malignant Solid Neoplasm
Recurrent Childhood Ependymoma
Recurrent Childhood Malignant Germ Cell Tumor
Recurrent Childhood Medulloblastoma
Recurrent Childhood Non-Hodgkin Lymphoma
Recurrent Childhood Osteosarcoma
Recurrent Childhood Rhabdomyosarcoma
Recurrent Childhood Soft Tissue Sarcoma
Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Neuroblastoma
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdoid Tumor
Refractory Childhood Malignant Germ Cell Tumor
Refractory Childhood Osteosarcoma
Refractory Childhood Rhabdomyosarcoma
Refractory Childhood Soft Tissue Sarcoma
Refractory Ependymoma
Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Wilms Tumor
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-101 (Larotrectinib) in Patients With Tumors Harboring Actionable NTRK Fusions NCT03213704
Disease/ConditionAdvanced Malignant Solid Neoplasm
Recurrent Ependymoma
Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Recurrent Glioma
Recurrent Hepatoblastoma
Recurrent Kidney Wilms Tumor
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Rhabdoid Tumor
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Refractory Ependymoma
Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Refractory Glioma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-292 in Patients With Tumors Harboring RET Gene Alterations NCT04320888
Disease/ConditionHematopoietic and Lymphatic System Neoplasm
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Hepatoblastoma
Recurrent Histiocytic and Dendritic Cell Neoplasm
Recurrent Langerhans Cell Histiocytosis
Recurrent Lymphoma
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Rhabdoid Tumor
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Recurrent WHO Grade 2 Glioma
Refractory Ependymoma
Refractory Ewing Sarcoma
Refractory Hepatoblastoma
Refractory Histiocytic and Dendritic Cell Neoplasm
Refractory Langerhans Cell Histiocytosis
Refractory Lymphoma
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Refractory WHO Grade 2 Glioma
Wilms Tumor
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice)- Phase 2 Subprotocol of LY3023414 in Patients With Solid Tumors NCT03213678
Disease/ConditionAdvanced Malignant Solid Neoplasm
Ann Arbor Stage III Non-Hodgkin Lymphoma
Ann Arbor Stage IV Non-Hodgkin Lymphoma
Malignant Glioma
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Glioma
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Solid Neoplasm
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Primary Central Nervous System Neoplasm
Rhabdoid Tumor
Stage III Osteosarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Osteosarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Stage IVA Osteosarcoma AJCC v7
Stage IVB Osteosarcoma AJCC v7
Wilms Tumor
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Tipifarnib in Patients With Tumors Harboring HRAS Genomic Alterations NCT04284774
Disease/ConditionMalignant Solid Neoplasm
Recurrent Adrenal Gland Pheochromocytoma
Recurrent Ectomesenchymoma
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Hepatoblastoma
Recurrent Kidney Wilms Tumor
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Glioma
Recurrent Medulloblastoma
Recurrent Melanoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Rhabdoid Tumor
Recurrent Rhabdoid Tumor of the Kidney
Recurrent Rhabdomyosarcoma
Recurrent Soft Tissue Sarcoma
Recurrent Thyroid Gland Carcinoma
Recurrent WHO Grade 2 Glioma
Refractory Adrenal Gland Pheochromocytoma
Refractory Ependymoma
Refractory Ewing Sarcoma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Glioma
Refractory Medulloblastoma
Refractory Melanoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Rhabdoid Tumor
Refractory Rhabdoid Tumor of the Kidney
Refractory Rhabdomyosarcoma
Refractory Soft Tissue Sarcoma
Refractory Thyroid Gland Carcinoma
Refractory WHO Grade 2 Glioma
Principal InvestigatorLisa Gennarini
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol NCT03155620
Disease/ConditionAdvanced Malignant Solid Neoplasm
Ann Arbor Stage III Non-Hodgkin Lymphoma
Ann Arbor Stage IV Non-Hodgkin Lymphoma
Histiocytic Sarcoma
Juvenile Xanthogranuloma
Langerhans Cell Histiocytosis
Malignant Glioma
Recurrent Childhood Rhabdomyosarcoma
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Glioma
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdoid Tumor
Recurrent Soft Tissue Sarcoma
Refractory Ewing Sarcoma
Refractory Glioma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Rhabdoid Tumor
Stage III Osteosarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Osteosarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Stage IVA Osteosarcoma AJCC v7
Stage IVB Osteosarcoma AJCC v7
Wilms Tumor
Principal InvestigatorAlice Lee
Trial DescriptionNCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol NCT03155620
Disease/ConditionAdvanced Malignant Solid Neoplasm
Ann Arbor Stage III Non-Hodgkin Lymphoma
Ann Arbor Stage IV Non-Hodgkin Lymphoma
Histiocytic Sarcoma
Juvenile Xanthogranuloma
Langerhans Cell Histiocytosis
Malignant Glioma
Recurrent Childhood Rhabdomyosarcoma
Recurrent Ependymoma
Recurrent Ewing Sarcoma
Recurrent Glioma
Recurrent Hepatoblastoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Solid Neoplasm
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Non-Hodgkin Lymphoma
Recurrent Osteosarcoma
Recurrent Peripheral Primitive Neuroectodermal Tumor
Recurrent Primary Central Nervous System Neoplasm
Recurrent Rhabdoid Tumor
Recurrent Soft Tissue Sarcoma
Refractory Ewing Sarcoma
Refractory Glioma
Refractory Hepatoblastoma
Refractory Langerhans Cell Histiocytosis
Refractory Malignant Germ Cell Tumor
Refractory Malignant Solid Neoplasm
Refractory Medulloblastoma
Refractory Neuroblastoma
Refractory Non-Hodgkin Lymphoma
Refractory Osteosarcoma
Refractory Peripheral Primitive Neuroectodermal Tumor
Refractory Primary Central Nervous System Neoplasm
Refractory Rhabdoid Tumor
Refractory Rhabdomyosarcoma
Rhabdoid Tumor
Stage III Osteosarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Osteosarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Stage IVA Osteosarcoma AJCC v7
Stage IVB Osteosarcoma AJCC v7
Wilms Tumor
Principal InvestigatorAlice Lee
Trial DescriptionNephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network NCT01209000
Disease/ConditionMinimal Change Disease (MCD)
Membranous Nephropathy
Glomerulosclerosis, Focal Segmental
Principal InvestigatorFrederick Kaskel
Trial DescriptionNephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network NCT01209000
Disease/ConditionMinimal Change Disease (MCD)
Membranous Nephropathy
Glomerulosclerosis, Focal Segmental
Principal InvestigatorFrederick Kaskel
Trial DescriptionNeuroblastoma Biology Studies NCT00904241
Disease/ConditionGanglioneuroblastoma
Localized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Regional Neuroblastoma
Stage 4 Neuroblastoma
Stage 4S Neuroblastoma
Principal InvestigatorLisa Gennarini
Trial 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 DescriptionNeuropsychological, Social, Emotional, and Behavioral Outcomes in Children With Cancer NCT00772200
Disease/ConditionChildhood Malignant Neoplasm
Principal InvestigatorLisa Gennarini
Trial DescriptionNMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO) NCT02679144
Disease/ConditionNeuroblastoma
Principal InvestigatorDaniel Weiser
Trial DescriptionNMTT- Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO) NCT02679144
Disease/ConditionNeuroblastoma
Principal InvestigatorDaniel Weiser
Trial DescriptionObservational Study of Pediatric Rheumatic Diseases: The CARRA Registry NCT02418442
Disease/ConditionRheumatic Joint Disease
Principal InvestigatorDawn Wahezi
Trial DescriptionONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study NCT05580562
Disease/ConditionH3 K27M
Glioma
Principal InvestigatorAdilia Hormigo
Trial DescriptionOptimize PRO Transcatheter Aortic Valve Replacement Post Market Study NCT04091048
Disease/ConditionSymptomatic Aortic Stenosis
Principal InvestigatorMohamed Azeem Latib
Trial DescriptionOptimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure? - randomized non blinded studyNCT04479735
Disease/ConditionPain, Procedural
Principal InvestigatorCourtney Mcnamara
Trial DescriptionOptimizing the Patient Experience: Virtual Reality Goggle Utilization for Venipuncture Distraction - Can we Decrease Anxiety and Pain During This Common Procedure? - randomized non blinded studyNCT04479735
Disease/ConditionPain, Procedural
Principal InvestigatorCourtney Mcnamara
Trial 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 DescriptionPazopanib Neoadjuvant Trial in Non-Rhabdomyosarcoma Soft Tissue Sarcomas (PAZNTIS): A Phase II/III Randomized Trial of Preoperative Chemoradiation or Preoperative Radiation Plus or Minus Pazopanib (NSC# 737754) NCT02180867
Disease/ConditionAlveolar Soft Part Sarcoma
Angiomatoid Fibrous Histiocytoma
Atypical Fibroxanthoma
Clear Cell Sarcoma of Soft Tissue
Epithelioid Malignant Peripheral Nerve Sheath Tumor
Epithelioid Sarcoma
Extraskeletal Myxoid Chondrosarcoma
Extraskeletal Osteosarcoma
Fibrohistiocytic Neoplasm
Fibrosarcoma
Inflammatory Myofibroblastic Tumor
Intimal Sarcoma
Leiomyosarcoma
Liposarcoma
Liver Embryonal Sarcoma
Low Grade Fibromyxoid Sarcoma
Low Grade Myofibroblastic Sarcoma
Malignant Cutaneous Granular Cell Tumor
Malignant Peripheral Nerve Sheath Tumor
Malignant Triton Tumor
Mesenchymal Chondrosarcoma
Myxofibrosarcoma
Myxoid Chondrosarcoma
Myxoinflammatory Fibroblastic Sarcoma
Nerve Sheath Neoplasm
PEComa
Pericytic Neoplasm
Plexiform Fibrohistiocytic Tumor
Sclerosing Epithelioid Fibrosarcoma
Skin Glomus Tumor
Stage IB Soft Tissue Sarcoma AJCC v7
Stage IIB Soft Tissue Sarcoma AJCC v7
Stage III Soft Tissue Sarcoma AJCC v7
Stage IV Soft Tissue Sarcoma AJCC v7
Synovial Sarcoma
Undifferentiated High Grade Pleomorphic Sarcoma of Bone
Principal InvestigatorLisa Gennarini
Trial DescriptionPediatric Hepatic Malignancy International Therapeutic Trial (PHITT) NCT03533582
Disease/ConditionChildhood Hepatocellular Carcinoma
Childhood Malignant Liver Neoplasm
Fibrolamellar Carcinoma
Hepatoblastoma
Hepatocellular Malignant Neoplasm, Not Otherwise Specified
Principal InvestigatorAlice Lee
Trial DescriptionPHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS - Phase 1 portion: The palbociclib plus irinotecan and temozolomide combination part of the study will comprise of a dose escalation cohort (following a rolling 6 design), a dose expansion cohort.
The palbociclib plus topotecan and cyclophosphamide will comprise of a dose determination cohort(following a modified rolling 6 design), a dose expansion cohort, and Neuroblastoma tumor -specific cohort.
Phase 2 open-label, randomized portion of the study will randomize patients in a 2:1 ratio to receive either palbociclib in combination with irinotecan and temozolomide (Arm A) or irinotecan and temozolomide chemotherapy alone (Arm B). Randomization will be stratified using block randomization by type and time of current disease recurrence (primary refractory or 1st recurrence < 2 years versus 1st recurrence ≥ 2 years or 2nd or greater recurrence).NCT03709680
Disease/ConditionEwing Sarcoma
Solid Tumors
Rhabdoid Tumor
Rhabdomyosarcoma
Neuroblastoma
Medulloblastoma
Diffuse Intrinsic Pontine Glioma
Principal InvestigatorAlice Lee
Trial DescriptionPhase 1-2 Trial of Gamunex (Intravenous Gammaglobulin) for Sickle Cell Acute Pain NCT01757418
Disease/ConditionSickle Cell Disease
Pain
Principal InvestigatorDeepa Manwani
Trial DescriptionPhase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis NCT05828069
Disease/ConditionRecurrent Langerhans Cell Histiocytosis
Refractory Langerhans Cell Histiocytosis
Principal InvestigatorAlice Lee
Trial DescriptionPhase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis NCT05828069
Disease/ConditionRecurrent Langerhans Cell Histiocytosis
Refractory Langerhans Cell Histiocytosis
Principal InvestigatorAlice Lee
Trial DescriptionPhase 2 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 III Multicenter Open-label Randomized Clinical Trial Comparing Everolimus and Low Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil at 6 mo Post-Transplant to Prevent Long-term Complications After Pediatric Heart Transplantation - Multicenter open-label randomized clinical trial with randomization within 4 strata, defined by donor-specific antibody status and center annual transplant volume. There are 2 parallel groups of equal sizes for randomization: everolimus/low-dose tacrolimus and tacrolimus/mycophenolate mofetil.NCT03386539
Disease/ConditionPediatric Heart Transplantation
Immunosuppression
Chronic Kidney Diseases
Cardiac Allograft Vasculopathy
Heart Transplant Failure and Rejection
Post-transplant Lymphoproliferative Disorder
Heart Transplant Infection
Principal InvestigatorDaphne Hsu
Trial DescriptionPhase III Randomized 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 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 DescriptionProspective Cohort Study to Evaluate Immunologic Response Following COVID-19 Vaccination in Children, Adolescents and Young Adults With Cancer NCT05228275
Disease/ConditionCOVID-19 Infection
Hematopoietic and Lymphatic System Neoplasm
Malignant Solid Neoplasm
Principal InvestigatorAlice Lee
Trial DescriptionRandomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial NCT03107871
Disease/ConditionCmv Congenital
CMV
Congenital Cmv
SNHL
Sensorineural Hearing Loss
Principal InvestigatorMichel Nassar
Trial DescriptionRandomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants: ValEAR Trial NCT03107871
Disease/ConditionCmv Congenital
CMV
Congenital Cmv
SNHL
Sensorineural Hearing Loss
Principal InvestigatorMichel Nassar
Trial DescriptionRandomized 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 DescriptionRandomized Study of Intravenous Calaspargase Pegol (SC-PEG Asparaginase) and Intravenous Oncaspar in Children and Adolescents With Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma NCT01574274
Disease/ConditionAcute Lymphoblastic Leukemia
Lymphoblastic Lymphoma
Principal InvestigatorLisa Gennarini
Trial DescriptionREal-World Analyses of Stroke - Thrombus Occlusion REtrieval NCT04451525
Disease/ConditionAcute Ischemic Stroke
Vessel Occlusion
Stroke
Ischemic
Principal InvestigatorNeil Haranhalli
Trial DescriptionRegistry of Asthma Patients Initiating DUPIXENT® (RAPID) NCT04287621
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionRegistry of Asthma Patients Initiating DUPIXENT® (RAPID) NCT04287621
Disease/ConditionAsthma
Principal InvestigatorGolda Hudes
Trial DescriptionRenal Tumors Classification, Biology, and Banking Study - PRIMARY OBJECTIVES:
I. Classify patients with renal tumors by histological categorization, surgico-pathological stage, presence of metastases, age at diagnosis, tumor weight, and loss of heterozygosity for chromosomes 1p and 16q, to define eligibility for a series of therapeutic studies.
(As of Amendment 8, this aim is limited to patients with institutionally classified Stage I to IV focal or diffuse anaplasia identified at initial biopsy or primary nephrectomy OR at delayed nephrectomy/second biopsy.)
II. To maintain a biological samples bank to make specimens available to scientists to evaluate additional potential biological prognostic variables and for the conduct of other research by scientists.
SECONDARY OBJECTIVES:
I. To monitor outcome for those patients who are not eligible for a subsequent therapeutic study.
II. To describe whether the pulmonary tumor burden correlates with outcome in Stage IV patients.(Completed as of Amendment 7)
III. To describe the sensitivity and specificity of abdominal computed tomography (CT) by comparison with surgical and pathologic findings for identification of local tumor spread beyond the renal capsule to adjacent muscle and organs, lymph node involvement at the renal hilum and in the retroperitoneum, preoperative tumor rupture and metastases to the liver. (Completed as of Amendment 7)
IV. To compare the sensitivity and specificity of preoperative abdominal CT and MRI for the identification and differentiation of nephrogenic rests and Wilms tumor in children with multiple renal lesions. (Completed as of Amendment 7)
V. To correlate the method of conception (natural versus assisted reproductive technology) with the development of Wilms tumor. (Completed as of Amendment 7)
VI. To evaluate the frequency of integrase interactor 1 (INI1) mutations in renal and extrarenal malignant rhabdoid tumor of the kidney and to determine the incidence of germline and inherited versus somatic mutations to facilitate clinical correlations on the companion study AREN0321. (Completed as of Amendment 7)
OUTLINE:
Tumor tissue, blood, and urine samples are collected for research studies, including immunohistochemistry. CT scans and MRIs are also performed. Loss of heterozygosity analyses (chromosome 1p and 16q) are performed by extraction of DNA. DNA polymorphisms are assayed by polymerase chain reaction using standard methodology. Leftover specimens are archived for future studies. (LOH and INI1 testing discontinued as of April 2014)
Patients are followed up periodically for 5 years.NCT00898365
Disease/ConditionAdult Cystic Nephroma
Anaplastic Kidney Wilms Tumor
Angiolipoma
Cellular Congenital Mesoblastic Nephroma
Classic Congenital Mesoblastic Nephroma
Clear Cell Sarcoma of the Kidney
Congenital Mesoblastic Nephroma
Cystic Partially Differentiated Kidney Nephroblastoma
Diffuse Hyperplastic Perilobar Nephroblastomatosis
Extrarenal Rhabdoid Tumor
Kidney Medullary Carcinoma
Kidney Neoplasm
Kidney Oncocytoma
Kidney Wilms Tumor
Metanephric Adenofibroma
Metanephric Adenoma
Metanephric Stromal Tumor
Metanephric Tumor
Mixed Congenital Mesoblastic Nephroma
Ossifying Renal Tumor of Infancy
Papillary Renal Cell Carcinoma
Renal Cell Carcinoma
Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions
Rhabdoid Tumor of the Kidney
Wilms Tumor
Principal InvestigatorLisa Gennarini
Trial DescriptionRenal Tumors Classification, Biology, and Banking Study - PRIMARY OBJECTIVES:
I. Classify patients with renal tumors by histological categorization, surgico-pathological stage, presence of metastases, age at diagnosis, tumor weight, and loss of heterozygosity for chromosomes 1p and 16q, to define eligibility for a series of therapeutic studies.
(As of Amendment 8, this aim is limited to patients with institutionally classified Stage I to IV focal or diffuse anaplasia identified at initial biopsy or primary nephrectomy OR at delayed nephrectomy/second biopsy.)
II. To maintain a biological samples bank to make specimens available to scientists to evaluate additional potential biological prognostic variables and for the conduct of other research by scientists.
SECONDARY OBJECTIVES:
I. To monitor outcome for those patients who are not eligible for a subsequent therapeutic study.
II. To describe whether the pulmonary tumor burden correlates with outcome in Stage IV patients.(Completed as of Amendment 7)
III. To describe the sensitivity and specificity of abdominal computed tomography (CT) by comparison with surgical and pathologic findings for identification of local tumor spread beyond the renal capsule to adjacent muscle and organs, lymph node involvement at the renal hilum and in the retroperitoneum, preoperative tumor rupture and metastases to the liver. (Completed as of Amendment 7)
IV. To compare the sensitivity and specificity of preoperative abdominal CT and MRI for the identification and differentiation of nephrogenic rests and Wilms tumor in children with multiple renal lesions. (Completed as of Amendment 7)
V. To correlate the method of conception (natural versus assisted reproductive technology) with the development of Wilms tumor. (Completed as of Amendment 7)
VI. To evaluate the frequency of integrase interactor 1 (INI1) mutations in renal and extrarenal malignant rhabdoid tumor of the kidney and to determine the incidence of germline and inherited versus somatic mutations to facilitate clinical correlations on the companion study AREN0321. (Completed as of Amendment 7)
OUTLINE:
Tumor tissue, blood, and urine samples are collected for research studies, including immunohistochemistry. CT scans and MRIs are also performed. Loss of heterozygosity analyses (chromosome 1p and 16q) are performed by extraction of DNA. DNA polymorphisms are assayed by polymerase chain reaction using standard methodology. Leftover specimens are archived for future studies. (LOH and INI1 testing discontinued as of April 2014)
Patients are followed up periodically for 5 years.NCT00898365
Disease/ConditionAdult Cystic Nephroma
Anaplastic Kidney Wilms Tumor
Angiolipoma
Cellular Congenital Mesoblastic Nephroma
Classic Congenital Mesoblastic Nephroma
Clear Cell Sarcoma of the Kidney
Congenital Mesoblastic Nephroma
Cystic Partially Differentiated Kidney Nephroblastoma
Diffuse Hyperplastic Perilobar Nephroblastomatosis
Extrarenal Rhabdoid Tumor
Kidney Medullary Carcinoma
Kidney Neoplasm
Kidney Oncocytoma
Kidney Wilms Tumor
Metanephric Adenofibroma
Metanephric Adenoma
Metanephric Stromal Tumor
Metanephric Tumor
Mixed Congenital Mesoblastic Nephroma
Ossifying Renal Tumor of Infancy
Papillary Renal Cell Carcinoma
Renal Cell Carcinoma
Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions
Rhabdoid Tumor of the Kidney
Wilms Tumor
Principal InvestigatorLisa Gennarini
Trial DescriptionRisk-Stratified Randomized Phase III Testing of Blinatumomab (NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL) NCT02101853
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionRisk-Stratified Randomized Phase III Testing of Blinatumomab (NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL) NCT02101853
Disease/ConditionRecurrent B Acute Lymphoblastic Leukemia
Principal InvestigatorAlice Lee
Trial DescriptionRisk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome NCT02521493
Disease/ConditionAcute Myeloid Leukemia
Down Syndrome
Myelodysplastic Syndrome
Myeloid Leukemia Associated With Down Syndrome
Myeloproliferative Neoplasm
Principal InvestigatorLisa Gennarini
Trial 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 - Adults: 240 mg IV (in a vein) over 30 minutes every 2 weeks OR 480 mg IV over 30 minutes every 4 weeks. Children and Adolescents weighing 40 kg or more: 240 mg IV over 30 minutes every 2 weeks OR 480 mg IV over 30 minutes every 4 weeks.
Children and Adolescents weighing less than 40 kg: 3 mg/kg IV over 30 minutes every 2 weeks. A maximum of 24 cycles will be given on study. Participants may continue to receive Nivolumab unless they develop serious side effects or the tumor worsens.
There were two parts to this study. The first part, Part A, was for patients who have relapsed or have progressive disease after their BMT. Part A is now closed. The second part, Part B, is for patients who have not yet relapsed or progressed after BMT.NCT03465592
Disease/ConditionSarcoma
Solid Tumor, Adult
Solid Tumor, Childhood
Principal InvestigatorDavid Loeb
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 DescriptionSurveillance HeartCare® Outcomes Registry NCT03695601
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionSurveillance HeartCare® Outcomes Registry NCT03695601
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionThe Collection of Research Samples and/or Data for Repository From Related or Unrelated Hematopoietic Stem Cell Transplantation Recipients for the National Marrow Donor Program NCT00495300
Disease/ConditionBone Marrow Transplantation
Principal InvestigatorIra Braunschweig
Trial DescriptionThe Collection of Research Samples and/or Data for Repository From Related or Unrelated Hematopoietic Stem Cell Transplantation Recipients for the National Marrow Donor Program NCT00495300
Disease/ConditionBone Marrow Transplantation
Principal InvestigatorIra Braunschweig
Trial DescriptionThe Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study NCT02402244
Disease/ConditionCarcinoma In Situ
Central Nervous System Neoplasm
Childhood Immature Teratoma
Childhood Langerhans Cell Histiocytosis
Childhood Mature Teratoma
Congenital Mesoblastic Nephroma
Desmoid Fibromatosis
Ganglioneuroma
Lymphoproliferative Disorder
Malignant Solid Neoplasm
Melanocytic Neoplasm
Myeloproliferative Neoplasm
Neuroendocrine Neoplasm
Stromal Neoplasm
Principal InvestigatorAlice Lee
Trial DescriptionThe Tether™ - Vertebral Body Tethering System Post Approval Study NCT04505579
Disease/ConditionScoliosis Idiopathic
Principal InvestigatorJaime Gomez
Trial DescriptionThe Tether™ - Vertebral Body Tethering System Post Approval Study NCT04505579
Disease/ConditionScoliosis Idiopathic
Principal InvestigatorJaime Gomez
Trial 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 Children With All Stages of Hepatoblastoma With Temsirolimus (NSC#683864) Added to High Risk Stratum Treatment NCT00980460
Disease/ConditionPRETEXT I Hepatoblastoma
PRETEXT II Hepatoblastoma
PRETEXT III Hepatoblastoma
PRETEXT IV Hepatoblastoma
Principal InvestigatorLisa Gennarini
Trial DescriptionTreatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents NCT03020030
Disease/ConditionAcute Lymphoblastic Leukemia, Pediatric
Principal InvestigatorLisa Gennarini
Trial DescriptionTreatment of Patients With Newly Diagnosed Standard Risk B-Lymphoblastic Leukemia (B-ALL) or Localized B-Lineage Lymphoblastic Lymphoma (B-LLy) NCT01190930
Disease/ConditionAcute Lymphoblastic Leukemia
Adult B Lymphoblastic Lymphoma
Ann Arbor Stage I B Lymphoblastic Lymphoma
Ann Arbor Stage II B Lymphoblastic Lymphoma
Childhood B Acute Lymphoblastic Leukemia
Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1
Childhood B Lymphoblastic Lymphoma
Down Syndrome
Hypodiploid B Acute Lymphoblastic Leukemia
Philadelphia Chromosome Positive
Principal InvestigatorLisa Gennarini
Trial DescriptionTrialNet Pathway to Prevention of T1D NCT00097292
Disease/ConditionDiabetes Mellitus, Type 1
Principal InvestigatorLisa Underland
Trial DescriptionTrialNet Pathway to Prevention of T1D NCT00097292
Disease/ConditionDiabetes Mellitus, Type 1
Principal InvestigatorLisa Underland
Trial DescriptionTrifecta-Heart cfDNA-MMDX Study: Comparing the DD-cfDNA Test to MMDx Microarray Test and Central HLA Antibody Test NCT04707872
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionTrifecta-Heart cfDNA-MMDX Study: Comparing the DD-cfDNA Test to MMDx Microarray Test and Central HLA Antibody Test NCT04707872
Disease/ConditionHeart Transplant Rejection
Principal InvestigatorSnehal Patel
Trial DescriptionTumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial NCT04589845
Disease/ConditionSolid Tumors
Principal InvestigatorHaiying Cheng
Trial 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 DescriptionUmbrella Long-Term Follow-Up Protocol NCT00736749
Disease/ConditionHematopoietic Cell Transplantation Recipient
Leukemia
Solid Tumor
Principal InvestigatorAlice Lee
Trial DescriptionUtilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients With Non-High-Risk Neuroblastoma NCT02176967
Disease/ConditionGanglioneuroblastoma
Localized Resectable Neuroblastoma
Localized Unresectable Neuroblastoma
Neuroblastoma
Principal InvestigatorAlice Lee
Trial 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