Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCT ID: NCT03368742

Last Updated: 2025-12-03

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-12-06
• Study Completion Date: 2026-10-15

Brief Summary

This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability 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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

SGT-001 - Dose Level 1

Single IV infusion of SGT-001 at starting dose

Group Type: EXPERIMENTAL

SGT-001

Intervention Type: GENETIC

AAV9 vector containing muscle-specific promoter and microdystrophin construct

SGT-001 - Dose Level 2

Single IV infusion of SGT-001 at next ascending dose

Group Type: EXPERIMENTAL

SGT-001

Intervention Type: GENETIC

AAV9 vector containing muscle-specific promoter and microdystrophin construct

Untreated Control

Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

SGT-001

AAV9 vector containing muscle-specific promoter and microdystrophin construct

Intervention Type: GENETIC

Eligibility Criteria

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

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Solid Biosciences Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Solid Bio Clinical Trials

Role: STUDY_DIRECTOR

Solid Biosciences

Locations

David Geffen School of Medicine at UCLA

Los Angeles, California, United States

University of Florida

Gainesville, Florida, United States

Countries

United States

References

Boehler JF, Brown KJ, Ricotti V, Morris CA. N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy. Skelet Muscle. 2024 Jan 16;14(1):2. doi: 10.1186/s13395-023-00334-y.

Reference Type: DERIVED

PMID: 38229112 (View on PubMed)

Other Identifiers

GX1001

Identifier Type: -

Identifier Source: org_study_id