Recruiting
Phase 3

SGT-003

Sponsor:

Solid Biosciences Inc.

Code:

NCT07160634

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: Male

Age: 7 - 11

Healthy Volunteers: Not accepted

Interventions

SGT-003

Placebo

Study Details

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Study ID

NCT07160634

Start date

Oct 22, 2025

Status verified date

Aug, 2026

Completion date

Jan, 2034

Anticipated

Primary completion date

Jan, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 7 - 11

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

80 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: SGT-003 in Part 1, Placebo in Part 2

Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and may receive a single IV infusion of matching Placebo in Part 2 per protocol-defined eligibility criteria.

experimental: Placebo in Part 1, SGT-003 in Part 2

Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2 per protocol-defined eligibility criteria.

Interventions

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

Primary outcome measure

  • Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 [ Time Frame: Baseline, Day 540 ]

Central Contacts and Locations

Central contacts

Solid Bio Clinical Trials

6173374680clinicaltrials@solidbio.com

Locations

Alberta Children's Hospital

Recruiting

Calgary, Alberta, Canada, T3B 6A8

Contacts

Israt Yasmeen

587-943-0851Israt.Yasmeen@ahs.ca

Principal Investigator:

Jean Mah, MD

BC Children's Hospital

Recruiting

Vancouver, British Columbia, Canada

Contacts

Nela Martic

604-875-2345nmartic3@cw.bc.ca

Principal Investigator:

Kathryn Selby, MD

The Hospital for Sick Children (SickKids)

Recruiting

Toronto, Ontario, Canada

Contacts

Ana Stosic

416-648-6831Ana.Stosic@SickKids.ca

Principal Investigator:

Hernan Gonorazky, MD

More Information

Sponsor

Solid Biosciences Inc.

Last update posted

Aug 13, 2026

Last verified

Aug, 2026

Keywords

  • SGT-003
  • Duchenne Muscular Dystrophy (DMD)
  • adeno-associated virus (AAV)
  • IMPACT DUCHENNE

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-05. This information was provided to ClinicalTrials.gov by Solid Biosciences Inc. on 2026-08-13.