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GC301 in infantile-onset Pompe disease
NCT05567627Phase 2Unknown
- Sponsor
- Seventh Medical Center of the People's Liberation Army General Hospital
- Start
- Aug 2022
- Primary completion
- Sep 2024
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of gc301 in Infantile-onset Pompe disease (IOPD), sponsored by Seventh Medical Center of the People's Liberation Army General Hospital. Primary endpoint: Safety and tolerability over time at 1 year. Status: unknown.
02
Drugs
gc301
03
Conditions
Infantile-onset Pompe disease (IOPD)
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