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
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.
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.
Parallel
RAG-17
DrugRAG-17 is a therapeutic small interfering RNA (siRNA).
Placebo
DrugPlacebo will be administered via intrathecal injection
Adverse Events(AEs)
Assesment of Safety and Tolerability: Incidence and severity of treatment-emergent Adverse Events(AEs) and Serious Adverse Events(SAEs)
Plasma Pharmacokinetic (PK) Parameter: AUC0-last
Area Under the Plasma Concentration-Time Curve from Time 0 to the Last Measurable Non-zero Concentration
Plasma Pharmacokinetic (PK) Parameter: AUC0-inf
Area Under the Plasma Concentration-Time Curve from Time 0 Extrapolated to Infinity
Plasma Pharmacokinetic (PK) Parameter: Cmax
Peak Plasma Concentration
Plasma Pharmacokinetic (PK) Parameter: Tmax
Time to reach Cmax. If the maximum value occurs at more than one time point, Tmax is defined as the first time point with this value
Click on the lead sponsor to view all of their trials.
