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
Ractigen Therapeutics.
32 participants
Dec 24, 2024
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
Inclusion Criteria6
- 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 Criteria5
- Documented p.F21C SOD1 mutation.
- 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.
- Current enrollment in any other interventional study.
- History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.
- Pregnant or currently breastfeeding.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
RAG-17 is a therapeutic small interfering RNA (siRNA).
Placebo will be administered via intrathecal injection
Locations(3)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06556394