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.
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.
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.
Parallel
SGT-003
DrugAdeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
Placebo
DrugIV infusion
Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540
Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540
Change From Baseline in 4-Stair Climb (4SC) Velocity (Tasks/s) at Day 540
Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540
Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540
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