What this Trial Is About

Researchers are conducting a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RAG-17 in patients with Amyotrophic Lateral Sclerosis (ALS) who have a Superoxide Dismutase Type 1 (SOD1) gene mutation. This study aims to understand how RAG-17, a therapeutic small interfering RNA (siRNA), behaves and affects this specific group of ALS patients. Participants will be assigned in a 3:1 ratio to receive either RAG-17 or a placebo administered via intrathecal injection. The dosing will be evaluated sequentially across different groups of participants using a rules-based design to assess the treatment's effects and safety. Placebo injections will mimic the administration method of RAG-17 to maintain study blinding. Throughout the study, researchers will monitor participants for adverse events before treatment and for 57 days after treatment. They will assess safety, tolerability, and how the drug moves through and acts within the body. Participants must provide informed consent and undergo various assessments during the study to ensure accurate evaluation of treatment effects and participant well-being.

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