Solid Biosciences SGT-001 Gene Therapy Phase 1/2 Clinical Trial (IGNITE DMD)

Active, Not RecruitingPhase 1/2Gene Therapy

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

Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks.

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

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

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Study Requirements and Criteria