Disease/ConditionOncology (Medical/Hematologic)
Pediatric Hematology/Oncology
Pediatrics
Principal InvestigatorLisa Gennarini, MD
Disease/ConditionGenetic Disorders
Genetics
Heart and Vascular Diseases
Liver Diseases
Pediatrics
Metabolic Diseases
Principal InvestigatorNadia Ovchinsky, MD
Trial DescriptionA Long-Term, Open-Label Follow-up Study of Tofacitinib for Treatment of Juvenile Idiopathic Arthritis (JIA) -
Click here for more information15-10-339-01
Disease/ConditionArthritis
Autoimmune Diseases
Musculoskeletal Diseases
Pediatrics
Rheumatic Diseases
Principal InvestigatorDawn Wahezi, MD
Trial DescriptionA Multicenter Single-arm Extension Study to Characterize the Long-term Safety of Cinacalcet Hydrochloride in the Treatment of Secondary Hyperparathyroidism in Pediatric Subjects With Chronic Kidney Disease on Dialysis -
Click here for more informationIRB# 20140159
Disease/ConditionDiabetes and Endocrine
Kidney Diseases (Chronic Kidney Disease)
Principal InvestigatorAnna Zolotnitskaya, MD
Trial DescriptionA Multicenter, Open-label, Randomized, Event-driven Study to Assess Efficacy, Safety and Pharmacokinetics of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension -
Click here for more information16-10-366-01
Disease/ConditionHeart and Vascular Diseases
Lung Diseases
Pediatrics
Pulmonary Hypertension
Principal InvestigatorNicole Sutton, MD
Trial DescriptionA Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Dose Optimization Study to Assess the Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome -
Click here for more information17-10-298-01
Disease/ConditionNeurodevelopmental Disorders
Pediatrics
Psychiatry - Non Cancer
Tourette Syndrome
Principal InvestigatorEric Hollander, MD
Trial DescriptionA Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease -
Click here for more information17-02-160Q-01
Disease/ConditionHematologic Diseases
Pediatrics
Sickle Cell
Principal InvestigatorCaterina Minniti, MD
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 -
Click here for more information16-10-374-01
Disease/ConditionPediatrics
Child Development
Endocrinology
Principal InvestigatorPing Zhou, MD
Trial DescriptionA Phase II Multi-Center, Randomized, Double-Blind, 24-Week, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy and Safety of RO5285119 in Children and Adolescents Age 5-17 with Autism Spectrum Disorder (ASD) -
Click here for more information16-10-117-01
Disease/ConditionAutism Spectrum Disorder
Neurodevelopmental Disorders
Pediatrics
Psychiatry - Non Cancer
Principal InvestigatorEric Hollander, MD
Trial DescriptionA Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated with Cholbam/Kolbam (Cholic Acid) -
Click here for more information17-08-442-01
Disease/ConditionGastroenterology & Liver Diseases
Pediatrics
Principal InvestigatorNadia Ovchinsky, MD
Trial DescriptionA Rando'd, Double-Blind, Placebo-Cont'd Trial to Evaluate Safety, Tolerability, PK and Efficacy of Single Infusion of Bezlotoxumab (MK-6072, Human Monoclonal Antibody to C.Diff Toxin B) in Children 1 to <18 Yrs Receiving Antibacterial Drug Tx for C.Diff -
Click here for more information17-10-212-01
Disease/ConditionInfectious Diseases
Pediatrics
Principal InvestigatorDavid Goldman, MD
Trial DescriptionA Randomized, Double-Blind, Placebo-Controlled, Multi-Center Pragmatic Clinical Trial To Evaluate The Effectiveness Of Low Dose Oral Methotrexate In Patients With Pediatric Crohn's Disease Initiating Anti-TNF Therapy (COMBINE) -
Click here for more information2017-7692
Disease/ConditionColitis and Crohn's Disease
Gastroenterology & Liver Diseases
Inflammatory Bowel Disease
Pediatrics
Principal InvestigatorGitit Tomer, MD
Trial DescriptionAn Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed with Early-Onset Idiopathic Bilateral Cataracts -
Click here for more information15-10-432-01
Disease/ConditionOphthalmic Diseases and Disorders
Pediatrics
Principal InvestigatorNorman Medow, MD
Disease/ConditionAutism Spectrum Disorder
Psychiatry - Non Cancer
Pediatrics
Principal InvestigatorEric Hollander, MD
Disease/ConditionAllergy & Immunology
Allergy and Immune Disorders
Asthma and other Respiratory Diseases
Pediatrics
Principal InvestigatorJonathan Feldman, PhD
Disease/ConditionPediatrics
Principal InvestigatorJudy Aschner, MD
Trial DescriptionEffectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity -
Click here for more information17-10-210-01
Disease/ConditionRehabilitation Medicine
Pediatrics
Principal InvestigatorYuxi Chen, MD
Disease/ConditionLeukemia
Pediatrics
Principal InvestigatorPeter Cole, MD
Disease/ConditionDiabetes and Endocrine
Principal InvestigatorRubina Heptulla, MD
Disease/ConditionCardiology
Cardiovascular and Thoracic Surgery
Heart and Vascular Diseases
Pediatrics
Principal InvestigatorJacqueline Lamour, MD
Disease/ConditionLung Diseases
Pediatrics
Asthma and other Respiratory Diseases
Principal InvestigatorDeepa Rastogi, MD
Trial DescriptionPhase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome -
Click here for more information2017-8076
Disease/ConditionPsychiatry - Non Cancer
Nervous System Diseases
Child Development
Pediatrics
Genetic Disorders
Principal InvestigatorEric Hollander, MD
Trial DescriptionPromoting Asthma Guidelines and Management through Technology-Based Intervention and Care Coordination (PRAGMATIC) -
Click here for more information2016-6258
Disease/ConditionAsthma and other Respiratory Diseases
Pediatrics
Lung Diseases
Principal InvestigatorMarina Reznik, MD
Trial DescriptionProtocol for a Research Sample Repository and for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies, and Marrow Toxic Injuries -
Click here for more information2015-5698
Disease/ConditionHematologic Diseases
Pediatrics
Neoplasms
Principal InvestigatorDavid Loeb, MD
Disease/ConditionBones, Muscles & Joints (Orthopaedics)
Orthopaedic Surgery
Pediatrics
Neoplasms
Musculoskeletal Diseases
Principal InvestigatorJacob Schulz, MD
Disease/ConditionHematologic Diseases
Pediatrics
Principal InvestigatorDavid Loeb, MD
Disease/ConditionPediatrics
Principal InvestigatorRubina Heptulla, MD
Trial DescriptionThe Pediatric Diabetes Consortium (PDC): Type 2 Diabetes in Youth Registry IRB# 2015-5480
Disease/ConditionDiabetes and Endocrine
Principal InvestigatorRubina Heptulla, MD
Trial DescriptionVALENS: A Ph 1/2, Random'd, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate Safety/Preliminary Efficacy of AT342, an AAV8-Delivered Gene Transfer Therapy in Crigler-Najjar Syndrome Subjects Aged 1 Yr and Older -
Click here for more information2017-7918
Disease/ConditionBrain and Neurological
Crigler-Najjar Syndrome
Gastroenterology & Liver Diseases
Heart and Vascular Diseases
Metabolic Diseases
Pediatrics
Principal InvestigatorNadia Ovchinsky, MD
Disease/ConditionAsthma and other Respiratory Diseases
Pediatrics
Lung Diseases
Principal InvestigatorDeepa Rastogi, MD