Overview

Safety and Tolerability of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

Status:
Enrolling by invitation

Trial end date:
2024-02-28

Target enrollment:

Participant gender:

Summary

The goal of this clinical trial is to evaluate the safety, tolerability and pharmacokinetics of RAG-17 in adult amyotrophic lateral sclerosis (ALS) patients with SOD1 mutation. Patients will receive drug treamtent via dose escalation which ranging from minimum of 60 mg to the maximum tolerated dose (MTD), after reaching the tolerated dose, a fixed dose of the drug is given once every two months for continuous treatment, and the total treatment cycle is 8 months. The duration of this study is two years.

Phase:

Early Phase 1

Details

Lead Sponsor:

Beijing Tiantan Hospital

Collaborator:

Ractigen Therapeutics