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

NCT07160634 | Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: SGT-003-3012025-522949-221013075
• Study Type: interventional
• Target: 80
• Locations: 2 countries
• Sites: 2

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

Keywords

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

Outcome Measures

Primary Outcomes (1)

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

  Baseline, Day 540

Secondary Outcomes (12)

• Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540

  Baseline, Day 540

• Change From Baseline in 4-Stair Climb (4SC) Velocity (tasks/s) at Day 540

  Baseline, Day 540

• Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540

  Baseline, Day 540

• Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540

  Baseline, Day 540

• Cumulative Loss of Function in NSAA Items at Day 540

  At Day 540

Study Arms (2)

SGT-003 followed by Placebo

EXPERIMENTAL

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

Drug: SGT-003; Drug: Placebo

Placebo followed by SGT-003

EXPERIMENTAL

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.

Drug: SGT-003; Drug: Placebo

Interventions

SGT-003 DRUG

Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)

Placebo followed by SGT-003; SGT-003 followed by Placebo

Placebo DRUG

IV infusion

Placebo followed by SGT-003; SGT-003 followed by Placebo

Eligibility Criteria

• Age: 7 Years - 11 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant is ambulatory.
• Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
• Negative for antibodies against adeno-associated virus.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Solid Biosciences Inc.: lead

Study Sites (2)

The Children's Hospital of Westmead

Sydney, New South Wales, Australia

RECRUITING

BC Children's Hospital

Vancouver, British Columbia, Canada

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Solid Bio Clinical Trials

CONTACT

6173374680; clinicaltrials@solidbio.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 29, 2025
• First Posted: September 8, 2025
• Study Start: October 22, 2025
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): January 1, 2034
• Last Updated: March 3, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); AU (1)