RecruitingPhase 1NCT06556394

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

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


Sponsor

Ractigen Therapeutics.

Enrollment

32 participants

Start Date

Dec 24, 2024

Study Type

INTERVENTIONAL

Conditions

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


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called RAG-17 for people with amyotrophic lateral sclerosis. The study is currently recruiting participants at 3 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGRAG-17

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

DRUGPlacebo

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

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NCT06556394


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