Phase 1 Study to Test Safety and Effects of AMX0114 Given by Spinal Injection in Adults With ALS

What this Trial Is About

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114, an investigational antisense oligonucleotide drug, in adults living with amyotrophic lateral sclerosis ALS. This phase I, placebo-controlled study aims to monitor how the drug affects ALS by targeting a protein linked to nerve cell damage, with the goal of understanding its impact on disease markers and overall safety. The study involves two groups one receiving AMX0114 and the other a placebo, both administered through intrathecal injection injection into the spinal canal once every 4 weeks for up to four doses on Days 1, 29, 57, and 85. Researchers will carefully observe participants throughout the dosing period to evaluate drug effects and potential side effects. Participants will undergo regular monitoring including physical and neurological exams, vital signs checks, laboratory tests, electrocardiograms, and biomarker assessments related to ALS progression. The primary focus is on safety and tolerability from Day 1 through Day 145. The study includes comprehensive evaluations to track drug behavior in the body and its influence on ALS-related biological markers over the study duration.

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis