Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Phase 1

Active, not recruiting

• Conditions: Duchenne Muscular Dystrophy

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

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.

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: 2025-08
• Last Update Submitted: 2025-08-01
• Last Update Posted: 2025-08-03
• 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

Primary Outcome Measures

Name	Time	Method
Number of Participants with Treatment Emergent Adverse Events (TEAEs)	Up to 5 years

Secondary Outcome Measures

Name	Time	Method
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters	Up to 5 years
Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants	Baseline, 12 months
Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants	Baseline, 12 months
Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score	Baseline, 12 months
Change from Baseline in Respiratory Function, as Measured by Forced Vital Capacity (FVC) % Predicted, Forced Expiratory Volume in 1 second (FEV1) % Predicted, and Peak Expiratory Flow (PEF) % Predicted	Baseline, 12 months
Change from Baseline in Ejection Fraction, As Measured by Echocardiography	Baseline,12 months
Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography	Baseline,12 months
Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography	Baseline,12 months
Change from Baseline in Quality of Life as Measured by the Paediatric Quality of Life Inventory (PedsQL) Duchenne muscular dystrophy (DMD) module and self-reported outcome measures as measured by the PODCI DMD module	Baseline, 12 months
Number of Participants with Clinically Significant Abnormalities in Vital Signs	Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Physical Examinations	Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)	Up to 5 years
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)	Baseline, 12 months
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)	Baseline, 12 months

Trial Locations

Locations (2)

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

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