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ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Sponsor: Atamyo Therapeutics
Summary
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
Official title: 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)
Key Details
Gender
All
Age Range
16 Years - 99 Years
Study Type
INTERVENTIONAL
Enrollment
6
Start Date
2022-09-01
Completion Date
2029-09-30
Last Updated
2025-10-06
Healthy Volunteers
No
Conditions
Interventions
ATA-100 (AAV9 encoding FKRP gene)
Single intravenous infusion on Day 0
Locations (3)
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