Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)
Active, Not RecruitingPhase 1/2Gene Therapy
Study Overview
- Age
- 4–17 years
- Phase
- Phase 1/2
- Sponsor
- Solid Biosciences
- Therapeutic Approach
- Gene Therapy
- Variant Requirement
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2017-12-06
- Primary Completion (Estimated)
- 2026-10-15
- Study Completion (Estimated)
- 2026-10-15
- Enrollment (Estimated)
- 12
- Countries
- United States
Study Requirements and Criteria
Steroid Use
Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks.
Inclusion Criteria
- 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
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
Clinical Trial Registry
NCT ID
NCT03368742This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.