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NCT07160634PHASE3Recruiting

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

Solid Biosciences Inc.

Start Date

10/22/2025

Completion Date

1/1/2034

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 Criteria

Age Range: 7 years to 11 years

Inclusion Criteria: * Participant is ambulatory. * Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype. * Negative for antibodies against adeno-associated virus. * On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice. * Meet 10-meter walk/run time criteria. * Meet time to rise from supine criteria. * Participant has bodyweight ≤50 kg. Exclusion Criteria: * Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy. * Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment. * Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report. Other Inclusion/Exclusion criteria to be applied as per protocol.

Interventions

DRUG

SGT-003

DRUG

Placebo

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Conditions

Duchenne Muscular Dystrophy

Locations

Arkansas Children's Hospital

Little Rock, Arkansas 72202

United States

Neurology Rare Disease Center

Flower Mound, Texas 75082

United States

Children's Hospital of the King's Daughters

Norfolk, Virginia 23510

United States

The Children's Hospital of Westmead

Sydney, New South Wales

Australia

Alberta Children's Hospital

Calgary, Alberta T3B 6A8

Canada

BC Children's Hospital

Vancouver, British Columbia

Canada