A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Prior treatment

• Treatment with dystrophin modifying drugs within 3 months prior to screening.
• Current or prior treatment with an approved or investigational gene transfer drug.
• Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.

Other

• Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.
  • Other inclusion or exclusion criteria apply.

• 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 mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
• Negative for AAV antibodies.
• Steroid regimen:
  • Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice.
  • Cohort 3: N/A
• Meet 10-meter walk/run time criteria
• Meet time to rise from supine criteria
• Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria
• Participant has body weight: ≤ 90 kg