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A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

RecruitingPhase 3

Sponsor: Solid Biosciences Inc.

Conditions: Duchenne Muscular Dystrophy

Interventions: SGT-003, Placebo

Countries: United States, Australia, Canada

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 overview

Sex: MALE

Age: 7 Years to 11 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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 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.

Locations (7)

Little Rock, Arkansas, United States
Flower Mound, Texas, United States
Norfolk, Virginia, United States
Sydney, New South Wales, Australia
Calgary, Alberta, Canada
Vancouver, British Columbia, Canada
Toronto, Ontario, Canada