A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Recruiting
Phase 1
Phase 2
Interventional Study
Duchenne Muscular Dystrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 0 – 17
- Sex
- Male
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 60 (estimated)
- Sponsor
- Solid Biosciences Inc. · Industry
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About This Trial
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 participant…
Trial Locations
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Eligibility Criteria
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 abi…
Contacts