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

Phase 1 Recruiting 32 enrolled Ractigen Therapeutics.

RandomizedParallel-groupDouble-blindTreatment

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

Timeline

Start: 2024-12-24
Primary completion: 2026-02
Completion: 2026-04

Drugs

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

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

Summary

Timeline

Drugs

Indications