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

What this Trial Is About

Researchers are studying the safety, tolerability, pharmacokinetics, and pharmacodynamics of a drug called RAG-17 in people with Amyotrophic Lateral Sclerosis (ALS) who have a specific genetic mutation in the Superoxide Dismutase Type 1 (SOD1) gene. This is a Phase 1 clinical trial designed to carefully evaluate these aspects in affected individuals. Participants are assigned to different groups to receive either RAG-17 or a placebo in a randomized, double-blind manner to ensure unbiased results. In this study, participants receive RAG-17 or placebo through an intrathecal injection, which delivers the treatment into the spinal fluid. The trial includes multiple dose levels tested sequentially across separate groups of participants. Each group receives the study drug or placebo at a 3:1 ratio, meaning three participants receive RAG-17 for every one participant receiving placebo. This setup helps researchers understand how the drug behaves in the body and monitor its effects. Participants will be closely monitored before and after treatment, with assessments including safety checks for adverse events up to 57 days after dosing. Blood and spinal fluid samples will be collected to study how the drug is processed and how long it stays in the body. The study includes detailed measurements of drug levels in plasma and cerebrospinal fluid at specific time points. Throughout the trial, the participants’ health and response to treatment will be evaluated to gather important information about RAG-17’s safety and behavior.

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