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

Purpose

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.

Condition

  • Duchenne Muscular Dystrophy

Eligibility

Eligible Ages
Between 0 Years and 17 Years
Eligible Sex
Male
Accepts Healthy Volunteers
No

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 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

Exclusion Criteria

  • 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. - Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing. Other inclusion or exclusion criteria apply.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Cohort 1: SGT-003 		All ambulatory participants from age 4 to < 7 years will receive a single IV infusion of SGT-003 on Day 1.
  • Genetic: SGT-003
    Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Experimental
Cohort 2: SGT-003 		All ambulatory participants from age 7 to < 12 years will receive a single IV infusion of SGT-003 on Day 1.
  • Genetic: SGT-003
    Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Experimental
Cohort 3: SGT-003 		All participants from age 0 to < 4 years will receive a single IV infusion of SGT-003 on Day 1.
  • Genetic: SGT-003
    Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Experimental
Cohort 4: SGT-003 		All ambulatory participants from age 12 to < 18 years will receive a single IV infusion of SGT-003 on Day 1.
  • Genetic: SGT-003
    Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Experimental
Cohort 5: SGT-003 		All non-ambulatory participants from age 10 to < 18 years will receive a single IV infusion of SGT-003 on Day 1.
  • Genetic: SGT-003
    Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)

Recruiting Locations

Washington University in St. Louis and nearby locations

Washington University in St. Louis
St Louis 4407066, Missouri 4398678 63110
Contact:
Natalie Goedeker
314-362-4919
neuromusclepediatricresearch@wustl.edu

More Details

NCT ID
NCT06138639
Status
Recruiting
Sponsor
Solid Biosciences Inc.

Study Contact

Solid Bio Clinical Trials
617-337-4680
clinicaltrials@solidbio.com