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

NCT ID: NCT07160634

Last Updated: 2025-12-12

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 80 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-10-22
• Study Completion Date: 2034-01-31

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 Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

SGT-003 followed by Placebo

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.

Group Type: EXPERIMENTAL

SGT-003

Intervention Type: DRUG

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

Placebo

Intervention Type: DRUG

IV infusion

Placebo followed by SGT-003

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.

Group Type: EXPERIMENTAL

SGT-003

Intervention Type: DRUG

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

Placebo

Intervention Type: DRUG

IV infusion

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)

Intervention Type: DRUG

Placebo

IV infusion

Intervention Type: DRUG

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.
• 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.

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Solid Biosciences Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

BC Children's Hospital

Vancouver, British Columbia, Canada

Site Status: RECRUITING

Countries

Canada

Central Contacts

Solid Bio Clinical Trials

Role: CONTACT

clinicaltrials@solidbio.com

6173374680

Facility Contacts

Nela Martic

Role: primary

nmartic3@cw.bc.ca

604-875-2345 ext. 6549

Other Identifiers

2025-522949-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

1013075

Identifier Type: OTHER

Identifier Source: secondary_id

SGT-003-301

Identifier Type: -

Identifier Source: org_study_id