A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Recruiting
Phase 3
Interventional Study
Duchenne Muscular Dystrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 7 – 11
- Sex
- Male
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 80 (estimated)
- Sponsor
- Solid Biosciences Inc. · Industry
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About This Trial
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 followe…
Trial Locations
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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.
* On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams p…
Contacts