Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

NCTID	NCT03368742 (View at clinicaltrials.gov)
Description	This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed. Participants currently enrolling are assigned to active treatment. Control participants enrolled under original protocol will continue through the study per the original protocol. (Show More)
Development Status	Inactive
Indication	Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term	DOID:11723
Compound Name	SGT-001
Compound Description	AAV9.CK8.Microdystrophin
Sponsor	Solid Biosciences Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	Micro-dystrophin
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	5E13 vg/kg
Dose 2	2E14 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	In September 2022, Solid Biosciences announced they would be pausing activities for SGT-001; "No longer developing" as of 2024 Annual Report