What is SGT-003 studying for Duchenne Muscular Dystrophy?

SGT-003 is being studied as a gene therapy for boys with Duchenne Muscular Dystrophy to evaluate its effects on muscle function and safety. This Phase 3 clinical trial by Solid Biosciences Inc. compares a single intravenous dose of SGT-003 to placebo to assess improvements in walking and muscle strength.

Who may be eligible for this Duchenne Muscular Dystrophy clinical trial?

Boys aged 7 to 11 years with ambulatory Duchenne Muscular Dystrophy who have a documented gene mutation may be eligible. Participants must be male, able to walk, and on a stable dose of prednisone or deflazacort. Please review eligibility criteria for more details.

Is there any cost to participate in this Duchenne Muscular Dystrophy clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel and related expenses may vary and are not guaranteed to be covered by the study.

Actively Recruiting

Phase 3

Age: 7Years - 11Years

MALE

ID07160634

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

Led by Solid Biosciences Inc. · Updated on 2026-07-16

Participants Needed

Research Sites

260 weeks

Total Duration

AI-Summary

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.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 7Years - 11Years

MALE

Eligibility Criteria

You may qualify if you...

Participant is ambulatory.
Confirmed clinical diagnosis of Duchenne Muscular Dystrophy with documented gene mutation.
Negative for antibodies against adeno-associated virus.
On a stable daily oral dose of at least 0.5 mg/kg/day prednisone or 0.75 mg/kg/day deflazacort for at least 6 months.
Meets 10-meter walk/run time criteria.
Meets time to rise from supine criteria.
Bodyweight is 50 kg or less.
Male participant aged between 7 and 11 years.

You will not qualify if you...

Current or prior treatment with approved or investigational gene transfer or gene editing therapy.
Use of vamorolone, givinostat, approved or investigational dystrophin or disease-modifying drugs, or other investigational drugs within 6 months or 5 half-lives before enrollment.
Diagnosis of Duchenne Muscular Dystrophy with deletion variants predicted not to express exons 1 to 11, 42 to 45, or 57 to 69 as documented by genetic report.

Research Team

Solid Bio Clinical Trials

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