ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5

NCTID	NCT05973630 (View at clinicaltrials.gov)
Description	The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2 dose-cohorts. (Show More)
Development Status	Active
Indication	Limb-Girdle Muscular Dystrophy, Type 2C/R5
Disease Ontology Term	DOID:0110277
Compound Name	ATA-200
Sponsor	Atamyo Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	6
Results Posted	Not Available

Target Gene/Variant	SGCG
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	AAV2/8
Editor Type	none
Dose 1	1.0E14 vg/kg
Dose 2	3.0E14 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States,Italy,France

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	US IND cleared 11/12/24