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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
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.
Age
7 - 11 years
Sex
MALE
Healthy Volunteers
No
The Children's Hospital of Westmead
Sydney, New South Wales, Australia
BC Children's Hospital
Vancouver, British Columbia, Canada
Start Date
October 22, 2025
Primary Completion Date
January 1, 2029
Completion Date
January 1, 2034
Last Updated
March 3, 2026
80
ESTIMATED participants
SGT-003
DRUG
Placebo
DRUG
Lead Sponsor
Solid Biosciences Inc.
NCT07037862
NCT06450639
NCT07129954
Data Source & Attribution
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