SGT-003: Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
Placebo: IV infusion
Study 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
Sex
MALE
Min age
7 Years
Max age
11 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 — Baseline, Day 540
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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