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Safety and Efficacy of RAG-21 in ALS with FUS Mutations
NCT07080801Phase 1Not yet recruiting
- Sponsor
- Beijing Tiantan Hospital
- Start
- Aug 2025
- Primary completion
- Aug 2026
- Modality
- Antisense DNA, Conjugated siRNA, MicroRNA (miRNA)
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Overview
Phase 1, open-label, single-arm trial of RAG-21 in amyotrophic lateral sclerosis (ALS), sponsored by Beijing Tiantan Hospital. Primary endpoint: Adverse Events and Serious Adverse Events at 197 days. Planned start Aug 2025; status: not yet recruiting.
02
Drugs
RAG-21
03
Conditions
amyotrophic lateral sclerosis (ALS)
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