Phase 3 Study Evaluating Single IV Dose of SGT-003 Gene Therapy in Boys Aged 7 to 11 with Duchenne Muscular Dystrophy

What this Trial Is About

Researchers are evaluating the efficacy and safety of a single intravenous infusion of SGT-003 gene therapy in ambulatory boys aged 7 to 11 with Duchenne Muscular Dystrophy DMD. This Phase 3, double-blind, placebo-controlled study aims to compare the effects of SGT-003 against placebo and includes long-term follow-up monitoring for at least five years after dosing. The study is designed to assess improvements in motor function and muscle protein levels in this pediatric population. Participants are randomly assigned to one of two groups one group receives SGT-003 first followed by placebo, while the other group receives placebo first followed by SGT-003. Each participant receives a single intravenous infusion during each part of the study. The trial includes two parts, each involving one infusion, and continues with long-term follow-up to monitor outcomes and safety. During the study, participants will undergo various assessments including measurements of motor function such as time to rise from supine position, stride velocity, stair climb speed, and walkingrunning speed. Muscle protein levels and respiratory function will also be evaluated. Safety will be monitored through adverse event tracking and heart function tests like ECG and echocardiography. Overall participation spans the two infusion periods plus a long-term follow-up of at least five years after receiving SGT-003.

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)