1539 | Term | Boston Children's Hospital

A Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia

This is a nonrandomized study of ruxolitinib in combination with a standard multi-agent chemotherapy regimen for the treatment of B-cell acute lymphoblastic leukemia. Part 1 of the study will optimize ...

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical ...

Bridge-Enhanced ACL Repair vs ACL Reconstruction

The goal of this trial is to compare the efficacy of the Bridge-Enhanced Anterior Cruciate Ligament Repair (BEAR™) technique with the current method of treatment for anterior cruciate ligament (ACL) ...

Study to Assess the Long-term Safety of Dupilumab Administered in Participants ≥6 Months to <18 Years of Age With Atopic Dermatitis (AD)

The primary objective of the study is to assess the long-term safety of dupilumab in pediatric participants with AD. The secondary objectives of the study are: * To assess the long-term efficacy of ...

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations ...

Immunotherapy of Relapsed Refractory Neuroblastoma With Expanded NK Cells

This NANT trial will determine the maximum tolerated dose (MTD) of autologous expanded natural killer (NK) cells when combined with standard dosing of dinutuximab and will assess the feasibility of ...

Preventing Asthma in High Risk Kids

This trial is a randomized, double-blind, placebo controlled trial designed to test whether two years treatment of preschool children aged 2-3 years of age at high risk for asthma with omalizumab ...

Riociguat in Children With Pulmonary Arterial Hypertension (PAH)

This study was designed to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of riociguat at age-, sex- and body-weight-adjusted doses of 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 ...

The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study

This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling ...

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations ...

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