Actively Recruiting
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
Led by Solid Biosciences Inc. · Updated on 2026-06-02
80
Participants Needed
5
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the effects of a gene therapy called SGT-003 in ambulant male children aged 7 to 11 years with Duchenne Muscular Dystrophy (DMD). This Phase 3, double-blind, placebo-controlled trial aims primarily to assess the impact of a single intravenous infusion of SGT-003 on the time it takes participants to rise from a supine position. Secondary goals include evaluating other efficacy and safety outcomes related to muscle function and respiratory measures. Participants will be randomly assigned to one of two groups: one receives SGT-003 first followed by placebo, and the other receives placebo first followed by SGT-003. Each participant will receive a single intravenous infusion during each of the two study parts. After these infusions, participants will be followed for at least five years in a long-term follow-up phase to monitor ongoing effects. Throughout the trial, participants will undergo assessments of muscle function, including walking and stair climbing speeds, and breathing capacity at baseline and after treatment periods. Researchers will also measure microdystrophin protein levels in tissue samples. Safety will be monitored through adverse event tracking and heart monitoring tests like electrocardiograms and echocardiograms. Overall participation will last several years to observe long-term outcomes.
CONDITIONS
Brief Title
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant is ambulatory.
- Established clinical diagnosis of Duchenne Muscular Dystrophy with documented gene mutation predictive of the 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 mg/kg/day deflazacort for at least 6 months before entering the study, with possible weight-based dose adjustments.
- Meets 10-meter walk/run time criteria.
- Meets time to rise from supine criteria.
- Bodyweight is 50 kilograms or less.
You will not qualify if you...
- Current or prior treatment with approved or investigational gene transfer or gene editing therapy.
- Exposure within 6 months or 5 half-lives (whichever is longer) prior to enrollment to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs, or other investigational drugs.
- Established DMD diagnosis associated with certain deletion variants not expressing exons 1 to 11, 42 to 45, or 57 to 69 as documented by genetic report.
- Other inclusion/exclusion criteria as per study protocol.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 540 days
Participants receive a single intravenous infusion of either the gene therapy SGT-003 or a matching placebo during each of the two parts of the study.
2 infusion visits and multiple assessment visits up to Day 540
Trial Site Locations
Total: 5 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Not Yet Recruiting
2
Neurology Rare Disease Center
Flower Mound, Texas, United States, 75082
Not Yet Recruiting
3
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States, 23510
Not Yet Recruiting
4
The Children's Hospital of Westmead
Sydney, New South Wales, Australia
Actively Recruiting
5
BC Children's Hospital
Vancouver, British Columbia, Canada
Actively Recruiting
Research Team
S
Solid Bio Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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