Actively Recruiting
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
Led by Ractigen Therapeutics. · Updated on 2025-01-08
32
Participants Needed
3
Research Sites
66 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
CONDITIONS
Official 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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Voluntarily consents to participate and provides written informed consent before any study procedures
- At least 18 years old at the time of consent
- Diagnosed with possible, laboratory supported probable, probable, or definite ALS per World Federation of Neurology El Escorial criteria
- Documented mutation in the SOD1 gene
- Forced vital capacity (FVC) 50% or greater of predicted value adjusted for sex, age, and height measured seated
- If taking riluzole or edaravone, must be on a stable dose for at least 30 days before Day 1 and expected to remain stable until study end
You will not qualify if you...
- Documented p.F21C SOD1 mutation
- Treated with another investigational drug, biological agent, or device within 1 month or 5 half-lives of that treatment, whichever is longer; specifically no prior small interfering RNA, stem cell, or gene therapy
- Currently enrolled in any other interventional study
- History of or positive test for HIV, hepatitis C antibody, or hepatitis B
- Currently pregnant or breastfeeding
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Beijing Tiantan Hospital
Beijing, China
Actively Recruiting
2
West China Hospital of Sichuan University
Chengdu, China
Actively Recruiting
3
The Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, China
Actively Recruiting
Research Team
L
Long-Cheng Li
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here