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

NCTID	NCT06138639 (View at clinicaltrials.gov)
Description	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 2 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. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up. (Show More)
Indication	Duchenne Muscular Dystrophy
Compound Name	SGT-003 (AAV-SLB101-CK8-microdystrophin containing R16-R17 nNOS binding domain)
Sponsor	Solid Biosciences Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	43

Target Gene/Variant	Micro-dystrophin
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Muscle cells
Delivery System	Viral transduction
Vector Type	AAV-SLB101
Editor Type
Dose 1	1 x 10^14 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
Recent Updates	FDA granted Rare Pediatric Disease Designation 4/1/24; topline data expected Q4 2024