Solid Biosciences Inc.
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
Inclusion Criteria: * Cohort 1: 4 to \<7 years of age * Cohort 2: 7 to \<12 years of age * Cohort 3: 0 to \< 4 years of age * Cohort 4: 12 to \< 18 years of age * Cohort 5: 10 to \< 18 years of age * Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in \< 30 seconds: * Cohorts 1, 2, and 4: Ambulatory * Cohort 3: Either ambulatory or non-ambulatory * Cohort 5: Non-ambulatory, but having been previously ambulatory by history * Established clinical diagnosis of DMD and documented dystrophin gene mut…
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)