Study of LTX-002, an Investigational Drug Delivered Into Spinal Fluid, in Adults With Amyotrophic Lateral Sclerosis

What this Trial Is About

Researchers are evaluating an investigational drug called LTX-002 in adults with amyotrophic lateral sclerosis ALS, a condition affecting movement, speech, and breathing. The study aims to assess the drugs safety, tolerability, how it moves through and affects the body, and its impact on indicators of ALS severity, including markers in blood and cerebrospinal fluid. This is a phase 12 trial conducted by Leal Therapeutics, Inc. Participants will receive LTX-002, an antisense oligonucleotide targeting SPTLC1 mRNA, through injections into the spinal fluid using a lumbar puncture. Several dose levels will be tested and compared with a placebo solution administered the same way. The study includes nine site visits, one overnight stay, and eight remote calls with staff. During the trial, participants will undergo medical history reviews, physical and neurological exams, electrocardiograms, lung function tests, muscle strength and speech assessments, and various questionnaires assessing ALS symptoms and mental health. Blood, urine, and cerebrospinal fluid samples will be collected for analysis. Safety and tolerability will be closely monitored through adverse events, lab tests, vital signs, and heart activity through Day 169 of the study.

A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis