Testing RAG-17 safety in people with ALS and a SOD1 gene mutation
Tests the drug RAG-17 against placebo in adults with ALS and a SOD1 gene change.
Plain-language title generated from the registry record
Registry title: 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
Key facts
- Where
- Beijing, Chengdu, and Hangzhou, ChinaSee the 3 sites
- Phase
- Phase 1
Where in China
- Beijing Tiantan HospitalBeijing, ChinaTaking people
- West China Hospital of Sichuan UniversityChengdu, ChinaTaking people
- The Second Affiliated Hospital Zhejiang University School of MedicineHangzhou, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Ractigen Therapeutics.
- Trial code
- NCT06556394
- Record updated
- 8 January 2025
- Original description
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 MutationRead full original descriptionShow less
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 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
Inclusion: Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures; ≥ 18 years of age at the time of informed consent; Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial; Documented SOD1 mutation; Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated); 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: 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; History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus; Documented p.F21C SOD1 mutation; Current enrollment in any other interventional study; Pregnant or currently breastfeeding.