Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.

ABSTRACT:

Importance

The etiology of amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease, is unknown. However, neuroinflammation and complement activation may play a role in disease progression.

Objective

To determine the effects of zilucoplan, an inhibitor of complement C5, in individuals with ALS.

Design, setting, and participants

Zilucoplan was tested as regimen A of the HEALEY ALS Platform Trial, a phase 2 to 3 multicenter, randomized, double-blind, placebo-controlled perpetual platform clinical trial with sharing of trial infrastructure and placebo data across multiple regimens. Regimen A was conducted from August 17, 2020, to May 4, 2022. A total of 162 participants were randomized to receive zilucoplan (122 [75.3%]) or regimen-specific placebo (40 [24.

SUBMITTER: Paganoni S 

PROVIDER: S-EPMC11833520 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature