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SGT-003 Gene Therapy for Duchenne Muscular Dystrophy

This study is testing a gene therapy called SGT-003 for boys with Duchenne Muscular Dystrophy (DMD) who can still walk. SGT-003 is designed to deliver a special gene to help with DMD. The study aims to see if SGT-003 helps improve how quickly boys can stand up from lying down. To join, boys must be between 7 and 11 years old, have a confirmed DMD diagnosis, and be taking a stable dose of prednisone. They also need to be negative for certain antibodies. This study plans to enroll 80 participants, but its current recruitment status is unclear.

Study design
This is a Phase 3, double-blind, placebo-controlled study. Participants will be randomly assigned to receive either SGT-003 or a placebo, then switch treatments later.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be monitored for at least 5 years after receiving SGT-003.

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NCT07160634

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

Recruiting
PHASE3Ages 7–11InterventionalTreatment
Solid Biosciences Inc.
~80 participants
Updated 2026-08-13 on ClinicalTrials.gov
What's tested:SGT-003Placebo

At a glance

Recruiting sites
4 of 7 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540
Measured over Baseline, Day 540

Conditions

Duchenne Muscular Dystrophy

Explore related trials

Duchenne Muscular DystrophyDMD Gene

Where it's being run

7 sites across 7 states
Arkansas1
Texas1
Virginia1
New South Wales1
Alberta1
British Columbia1
Ontario1

Who to contact

Solid Bio Clinical Trials
[email protected]
6173374680
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Participant is ambulatory.
Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
Negative for antibodies against adeno-associated virus serotype 9 (AAV9).
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

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.

What this trial measures

  • Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540Baseline, Day 540