Actively Recruiting
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Led by Solid Biosciences Inc. · Updated on 2026-05-08
80
Participants Needed
2
Research Sites
427 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 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 mg/kg/day deflazacort for at least 6 months prior to study entry.
- Meet 10-meter walk/run time criteria.
- Meet time to rise from supine criteria.
- Participant has bodyweight 50 kg.
You will not qualify if you...
- Current or prior treatment with approved or investigational gene transfer or gene editing therapies.
- Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs, or other investigational drugs within 6 months or 5 half-lives before enrollment.
- Established DMD diagnosis with deletions or variants predicted not to express exons 1-11, 42-45, or 57-69 of the DMD gene.
- Other inclusion/exclusion criteria as per protocol.
AI-Screening
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Trial Site Locations
Total: 2 locations
1
The Children's Hospital of Westmead
Sydney, New South Wales, Australia
Actively Recruiting
2
BC Children's Hospital
Vancouver, British Columbia, Canada
Actively Recruiting
Research Team
S
Solid Bio Clinical Trials
CONTACT
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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