NCT07160634 A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
| NCT ID | NCT07160634 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Solid Biosciences Inc. |
| Condition | Duchenne Muscular Dystrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 80 participants |
| Start Date | 2025-10-22 |
| Primary Completion | 2029-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 80 participants in total. It began in 2025-10-22 with a primary completion date of 2029-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 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.
Eligibility Criteria
Inclusion Criteria: * 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 Criteria: * 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07160634 clinical trial?
This trial is open to male participants only, aged 7 Years or older, up to 11 Years, studying Duchenne Muscular Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07160634 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 80 participants.
Is NCT07160634 currently recruiting?
Yes, NCT07160634 is actively recruiting participants. Contact the research team at clinicaltrials@solidbio.com for enrollment information.
Where is the NCT07160634 trial being conducted?
This trial is being conducted at Little Rock, United States, Flower Mound, United States, Norfolk, United States, Sydney, Australia and 3 additional locations.
Who is sponsoring the NCT07160634 clinical trial?
NCT07160634 is sponsored by Solid Biosciences Inc.. The trial plans to enroll 80 participants.