Search Results

• A Study Testing the Combo of Dasatinib or Imatinib to Chemo Treatment W/ Blinatumomab for Children, Adolescents, & YA W/ Ph+ or Ph-Like B-cell B-ALL

  A Study Testing the Combination of Dasatinib or Imatinib to Chemotherapy Treatment With Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or ABL-Class Philadelphia Chromosome-Like (Ph-Like) B-cell Acute Lymphoblastic Leukemia (B-ALL)

• A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/​or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations

  AAML1831 - A 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

• A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency

  A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)

• Endoscopic versus Shunt Treatment of Hydrocephalus in Infants (ESTHI)

  Endoscopic versus Shunt Treatment of Hydrocephalus in Infants (ESTHI)

• DAY101 vs. Standard of Care Chemotherapy in Pediatric Patients With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)

  LOGGIC/FIREFLY-2: A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients With Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy

• A RETROSPECTIVE CONTINUATION STUDY IN SMALL FIBER NEUROPATHY IN CHILDREN: PRESENTATION & CLINICAL FEATURES

  A RETROSPECTIVE CONTINUATION STUDY IN SMALL FIBER NEUROPATHY IN CHILDREN: PRESENTATION & CLINICAL FEATURES

• Beat Childhood Cancer Specimen Banking and Data Registry

  Specimen Banking With Clinical and Genomic Data Registry With the Establishment of Tumor Models for Pediatric Cancers

• A Retrospective Assessment of Novel Biomarkers Indicating Intestinal Inflammation and Intestinal Permeability, Identified During Non-Invasive Leaky Gut Testing

  A Retrospective Assessment of Novel Biomarkers Indicating Intestinal Inflammation and Intestinal Permeability, Identified During Non-Invasive Leaky Gut Testing

• A 4-Week, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Assess the Safety and Efficacy of Tenapanor for the Treatment of Irritable Bowel Syndrome with Constipation (IBS-C) in Pediatric Patients 6 to Less than 12 Years Old

  A 4-Week, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Assess the Safety and Efficacy of Tenapanor for the Treatment of Irritable Bowel Syndrome with Constipation (IBS-C) in Pediatric Patients 6 to Less than 12 Years Old

• Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

  Phase II Trial of Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma