A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Recruiting now · Phase 3 · Has a placebo group
Conditions studied: Duchenne Muscular Dystrophy
In brief
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.
Key facts
- Study ID
- NCT07160634
- Run by
- Solid Biosciences Inc.
- People needed
- 80
- Starts
- 2025-10-22
- Expected to finish
- 2034-01-01
- Last updated by the study team
- 2026-07-16
Who can join
Age: 7 and older, up to 11. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- The Children's Hospital of Westmead — Sydney, New South Wales, Australia (enrolling)
- Alberta Children's Hospital — Calgary, Alberta, Canada (enrolling)
- BC Children's Hospital — Vancouver, British Columbia, Canada (enrolling)
- Arkansas Children's Hospital — Little Rock, Arkansas, United States
- Neurology Rare Disease Center — Flower Mound, Texas, United States
- Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
Full record on ClinicalTrials.gov
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