Evaluating Tofersen Treatment Safety and Effects in Adults with ALS Without SOD1 or FUS Gene Mutations

What this Trial Is About

Researchers are evaluating the safety and biological effects of tofersen in adults who have amyotrophic lateral sclerosis ALS without mutations in the SOD1 gene. The study focuses on whether tofersen reduces levels of neurofilament light chain NfL in the blood and cerebrospinal fluid CSF, how well participants tolerate the treatment, and its impact on clinical outcomes and quality of life. Tofersen is already approved for treating SOD1-ALS, and this trial aims to explore its effects in non-SOD1 ALS cases. Participants will receive 100 mg of tofersen via lumbar puncture at eight time points over 24 weeks Weeks 0, 2, 4, 8, 12, 16, 20, and 24. After the dosing period, participants will have two follow-up visits at Weeks 28 and 32. The study involves only one group receiving tofersen, with no placebo or comparison groups. During the trial, participants will complete various questionnaires and undergo outcome assessments such as strength and breathing tests. Researchers will monitor safety and tolerability, measure changes in neurofilament levels, and track clinical and quality-of-life outcomes. The primary measurement is the proportion of participants with at least a 30% reduction in plasma NfL from baseline to Week 28, with additional safety monitoring continuing through study completion around eight months.

Tofersen in Non-SOD1 ALS