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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

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.