Status:
RECRUITING
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Lead Sponsor:
Solid Biosciences Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
7-11 years
Phase:
PHASE3
Brief 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...
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.
- 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.
- Other Inclusion/Exclusion criteria to be applied as per protocol.
Key Trial Info
Start Date :
October 22 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 1 2034
Estimated Enrollment :
80 Patients enrolled
Trial Details
Trial ID
NCT07160634
Start Date
October 22 2025
End Date
January 1 2034
Last Update
December 12 2025
Active Locations (1)
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1
BC Children's Hospital
Vancouver, British Columbia, Canada