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 and tolerability 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. (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 (ACTUAL)
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 (n=3)
Dose 2	2E14 vg/kg (n=9)
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