A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Study Details
Study Description
Brief Summary
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single IV infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study, dosed sequentially. Cohort 1 will include participants 4 to <6 years of age, inclusive. Cohort 2 will only be opened after dosing and monitoring a subset of participants in Cohort 1. Cohort 2 will include participants 6 to <8 years of age, inclusive. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Cohort 1: SGT-003 All participants 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 participants 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)
|
Outcome Measures
Primary Outcome Measures
- Incidence of treatment-emergent adverse events (AEs) [Day 360]
Secondary Outcome Measures
- Change from baseline in microdystrophin protein levels [Day 90, Day 360]
Microdystrophin expression evaluation in muscle biopsies
- Change from baseline in North Star Ambulatory Assessment (NSAA) total score [Day 360]
Assessment of muscle function using a 17-item scale
- Change from baseline in stride velocity 95th centile (SV95C) [Day 360]
Assessment of peak ambulatory performance captured by wearable activity monitoring device
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Cohort 1: 4 to <6 years of age, inclusive
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Cohort 2: 6 to <8 years of age, inclusive
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Ambulatory as defined as "being able to walk without the use of an assistive device."
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Negative for AAV antibodies.
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On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone or 0.75 mg/kg/day deflazacort for ≥12 weeks prior to entering the study.
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Cohort 1: <18 kg body weight
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Cohort 2: <30 kg body weight
Exclusion Criteria:
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Current or prior treatment with approved or investigational dystrophin modifying drugs such as eteplirsen, golodirsen, casimersen, and viltolarsen.
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Current or prior treatment with an approved or investigational gene transfer drug.
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Exposure to another investigational drug within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of California, Los Angeles Medical Center | Los Angeles | California | United States | 90095 |
| 2 | Nationwide Children's Hospital | Columbus | Ohio | United States | 43215 |
Sponsors and Collaborators
- Solid Biosciences Inc.
Investigators
- Study Director: Solid Bio Clinical Trials, Solid Biosciences
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- SGT-003-101