Status:
ACTIVE_NOT_RECRUITING
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Lead Sponsor:
Solid Biosciences Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
4-17 years
Phase:
PHASE1
PHASE2
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 r...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
December 6 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 15 2026
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT03368742
Start Date
December 6 2017
End Date
October 15 2026
Last Update
December 3 2025
Active Locations (2)
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1
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095
2
University of Florida
Gainesville, Florida, United States, 32610