Evaluating RAG-17 for ALS patients with SOD1 mutation

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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
• Email: lilc@ractigen.com
• Phone: +86 18051622388

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.