Status:
ACTIVE_NOT_RECRUITING
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Lead Sponsor:
Atamyo Therapeutics
Conditions:
LGMDR9
Eligibility:
All Genders
16-99 years
Phase:
PHASE1
Brief Summary
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
Detailed Description
Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene. This study is an open-label dos...
Eligibility Criteria
Inclusion
- 1\. Female and male ambulant patients
- 2\. Patients ≥ 16 years old
- 3\. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
- 4\. Moderate diaphragmatic muscle impairment
Exclusion
- 1\. Detectable serum neutralizing antibodies against AAV9
- 2\. Cardiomyopathy
Key Trial Info
Start Date :
September 1 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 30 2029
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT05224505
Start Date
September 1 2022
End Date
September 30 2029
Last Update
October 6 2025
Active Locations (3)
Enter a location and click search to find clinical trials sorted by distance.
1
Rigshospitalet, University of Copenhagen Blegdamsvej 9
Copenhagen, Denmark, 2100
2
Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital
Paris, France, 75013
3
Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing
Newcastle upon Tyne, United Kingdom, NE1 4LP