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

PHASE1RecruitingINTERVENTIONAL
Enrollment

32

Participants

Timeline

Start Date

December 24, 2024

Primary Completion Date

February 28, 2026

Study Completion Date

April 30, 2026

Conditions
Amyotrophic Lateral Sclerosis
Interventions
DRUG

RAG-17

RAG-17 is a therapeutic small interfering RNA (siRNA).

DRUG

Placebo

Placebo will be administered via intrathecal injection

Trial Locations (3)

Unknown

RECRUITING

Beijing Tiantan Hospital, Beijing

RECRUITING

West China Hospital of Sichuan University, Chengdu

RECRUITING

The Second Affiliated Hospital Zhejiang University School of Medicine, Hangzhou

All Listed Sponsors
lead

Ractigen Therapeutics.

OTHER