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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation
Sponsor: Ractigen Therapeutics.
Summary
This is a Phase 1, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability,Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
32
Start Date
2024-12-24
Completion Date
2026-04
Last Updated
2025-01-08
Healthy Volunteers
No
Conditions
Interventions
RAG-17
RAG-17 is a therapeutic small interfering RNA (siRNA).
Placebo
Placebo will be administered via intrathecal injection
Locations (3)
Beijing Tiantan Hospital
Beijing, China
West China Hospital of Sichuan University
Chengdu, China
The Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, China