A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)

Study Summary

Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up

Interventions

Study Locations

Facility	City	State	Country
Rigshospitalet, University of Copenhagen Blegdamsvej 9	Copenhagen		Denmark
Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital	Paris		France
Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing	Newcastle upon Tyne		United Kingdom

Official Trial Information

View on ClinicalTrials.gov