Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Phase 1

Active, not recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Genetic: SGT-001

• Registration Number: NCT03368742
• Lead Sponsor: Solid Biosciences Inc.

Brief Summary

This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.

The protocol was amended to drop the control arm after 4 participants were dosed. Participants currently enrolling are assigned to active treatment. Control participants enrolled under original protocol will continue through the study per the original protocol.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-12-05
• Study Start: 2017-12-06
• Study First Submitted QC: 2017-12-08
• Study First Posted: 2017-12-11
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-11
• Last Update Posted: 2025-04-16
• Primary Completion: 2026-10-15
• Study Completion: 2026-10-15

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 12

Inclusion Criteria

• Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
• Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
• Anti-AAV9 antibodies below protocol-specified thresholds
• Stable cardiac and pulmonary function
• Adolescents: non-ambulatory by protocol-specified criteria
• Children: ambulatory by protocol-specified criteria
• Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks

Exclusion Criteria

• Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results
• Abnormal liver function
• Abnormal renal function
• Clinically significant coagulation abnormalities
• Impaired cardiovascular function based on cardiac MRI or ECHO
• Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support
• Significant spinal deformity or presence of spinal rods
• Body mass index ≥ 95th percentile for age
• Exposure to another investigational drug within 3 months or 5 half-lives prior to screening
• Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening

Additional inclusion/exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
SGT-001 - Dose Level 2	SGT-001	Single IV infusion of SGT-001 at next ascending dose
SGT-001 - Dose Level 1	SGT-001	Single IV infusion of SGT-001 at starting dose

Primary Outcome Measures

Name	Time	Method
Primary safety endpoint	Baseline to 5 years	Incidence of adverse events

Secondary Outcome Measures

Name	Time	Method
Secondary safety endpoint	Baseline to 5 years	Incidence of abnormalities on Electrocardiogram (ECG)
Secondary efficacy endpoint	Baseline to 12 months	Changes from baseline at 12 months in quality of life as measured by self-reported outcome measures as measured by the Pediatric Outcomes Data Collection Instrument (PODCI) DMD module

Trial Locations

Locations (2)

David Geffen School of Medicine at UCLA; 🇺🇸 Los Angeles, California, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States