Evaluating RAG-17 for ALS patients with SOD1 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

This study is testing a new drug called RAG-17 to see if it is safe and effective for people with ALS who have a specific SOD1 gene mutation.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 32 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Ractigen Therapeutics. (Academic / other)
• Locations: 3 sites (Beijing and 2 other locations)
• Trial ID: NCT06556394 on ClinicalTrials.gov

What this trial studies

This Phase 1 clinical trial is designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of RAG-17 in individuals diagnosed with Amyotrophic Lateral Sclerosis (ALS) who have a specific mutation in the SOD1 gene. The study employs a randomized, double-blind, placebo-controlled methodology, where participants will receive either RAG-17 or a placebo in a 3:1 ratio. The trial will evaluate different dose levels across separate cohorts to determine the optimal dosing and safety profile of the drug.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of ALS and a documented SOD1 gene mutation.

Not a fit: Patients with the p.F21C SOD1 mutation or those currently receiving other investigational treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option for ALS patients with the SOD1 mutation.

How similar studies have performed: While there have been studies targeting ALS, this specific approach with RAG-17 in SOD1 mutation patients is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

1. Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.
2. ≥ 18 years of age at the time of informed consent.
3. Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.
4. Documented SOD1 mutation.
5. Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated).
6. If taking riluzole or edaravone, subject must be on a stable dose or ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.

Exclusion Criteria:

1. Documented p.F21C SOD1 mutation.
2. Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.
3. Current enrollment in any other interventional study.
4. History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.
5. Pregnant or currently breastfeeding.

Where this trial is running

Beijing and 2 other locations

• Beijing Tiantan Hospital — Beijing, China (Recruiting)
• West China Hospital of Sichuan University — Chengdu, China (Recruiting)
• The Second Affiliated Hospital Zhejiang University School of Medicine — Hangzhou, China (Recruiting)

Study contacts

• Study coordinator: Long-Cheng Li

Conditions Amyotrophic Lateral Sclerosis