SGT-003-301
Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
IRAS ID
1013075
Contact name
Kelsey Genda
Contact email
Sponsor organisation
Solid Biosciences Inc.
Clinicaltrials.gov Identifier
Research summary
SGT-003-301 is being conducted at multiple locations and will include participants from different regions. The trial is designed to be randomized, meaning participants are randomly assigned to different groups, and double-blind, meaning neither the participants nor the researchers know who is receiving the actual treatment or the placebo (a substance with no therapeutic effect).
The main goal of the study is to see how effective the drug SGT-003 is compared to a placebo in boys who can walk and have Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. The study will also assess other measures of effectiveness and safety.
Around 80 boys will take part in the study. They will be randomly assigned to one of two groups: one group will receive SGT-003 first and then switch to the placebo, while the other group will receive the placebo first and then switch to SGT-003.
The primary analysis will focus on the results from the first part of the study, after all participants have completed their assessments on Day 540. A final analysis will be done once all participants have completed the entire study.REC name
London - West London & GTAC Research Ethics Committee
REC reference
26/LO/0018
Date of REC Opinion
24 Feb 2026
REC opinion
Further Information Favourable Opinion