Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

Phase 0 Not yet recruiting 10 enrolled Beijing Tiantan Hospital

NaSingle-groupOpen-labelTreatment

Summary

This study primarily evaluates the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting.

Timeline

Start: 2025-07
Primary completion: 2026-12-31
Completion: 2026-12-31

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	RAG-17	Small interfering RNA (siRNA)	150 mg	Intrathecal

Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

Summary

Timeline

Drugs

Indications