RecruitingPhase 3NCT07160634

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

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy


Sponsor

Solid Biosciences Inc.

Enrollment

80 participants

Start Date

Oct 22, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.


Eligibility

Sex: MALEMin Age: 7 YearsMax Age: 11 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called SGT-003 for people with duchenne muscular dystrophy. The study is currently recruiting participants at 5 locations. People eligible for this study include men aged 7 Years to 11 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGSGT-003

Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)

DRUGPlacebo

IV infusion


Locations(6)

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Neurology Rare Disease Center

Flower Mound, Texas, United States

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

The Children's Hospital of Westmead

Sydney, New South Wales, Australia

Alberta Children's Hospital

Calgary, Alberta, Canada

BC Children's Hospital

Vancouver, British Columbia, Canada

View Full Details on ClinicalTrials.gov

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NCT07160634


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