Search Results

• A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma

  A 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)

• DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma

  Phase II Trial of Eflornithine/DFMO as Maintenance Therapy for Molecular High Risk/Very High Risk and Relapsed/Refractory Medulloblastoma

• A Study to Evaluate Etrasimod Treatment in Adolescents With Ulcerative Colitis

  An Open-Label, Single-Arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Etrasimod in Adolescent Subjects with Moderately to Severely Active Ulcerative Colitis

• A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

  4202-HEM-301: An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)

• A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

  A Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

• Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy

  A 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

• Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

  A Study Using Molecular Guided Therapy With Induction Chemotherapy Followed by a Randomized Controlled Trial of Standard Immunotherapy With or Without DFMO Followed by DFMO Maintenance for Subjects With Newly Diagnosed High-Risk Neuroblastoma

• Cystic Fibrosis National Registry

  Cystic Fibrosis National Registry

• Improving Care of Children with Complex Congenital Heart Disease

  National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children with Complex Congenital Heart Disease

• The Association of Kawasaki Disease and COVID-19 with Cardiac Complications:

  The Association of Kawasaki Disease and COVID-19 with Cardiac Complications: A Focused Sub-study of the International Kawasaki Disease Registry (IKDR)