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ASP2016 gene therapy in Friedreich Ataxia cardiomyopathy
NCT06483802Phase 1Withdrawn
- Sponsor
- Astellas Gene Therapies
- Start
- Nov 2024
- Primary completion
- Oct 2025
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
01
Overview
Phase 1, open-label, single-arm trial of ASP2016 in Friedreich ataxia (FA), sponsored by Astellas Gene Therapies. Primary endpoints: Treatment Emergent Adverse Events at 60 months, Serious Adverse Events at 60 months and 3 more. Planned start Nov 2024; status: withdrawn.
02
Drugs
ASP2016
03
Conditions
Friedreich ataxia (FA)
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