Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy
PhasePhase 1/Phase 2
Lead SponsorSolid Biosciences
StatusActive, not recruiting
Indication/ConditionDuchenne Muscular Dystrophy
This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Patients 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 subjects were dosed. Subjects currently enrolling are assigned to active treatment. Control subjects enrolled under original protocol will continue through the study per the original protocol.
Single IV infusion of SGT-001 at starting dose
Single IV infusion of SGT-001 at next ascending dose
Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.
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 patients) 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 wks Exclusion Criteria: Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect subject 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. Patients over 30 kg will not be eligible for treatment at this time. A weight limit of ≤ 18 kg will be implemented for the next two patients to be dosed.