A Phase 1-2, Open-label Study to Evaluate the Safety of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients With Gamma-sarcoglycanopathy (LGMDR5)

Study Summary

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).

Interventions

Study Locations

Facility	City	State	Country
Child Health Research Institute	Gainsville	Florida	United States

Official Trial Information

View on ClinicalTrials.gov