Efficacy and Safety of Ravulizumab, a Complement C5 Inhibitor, in Adults With Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.

ABSTRACT:

Importance

Additional therapies for amyotrophic lateral sclerosis (ALS) are urgently needed. Immune-mediated complement activation may be involved in ALS pathogenesis as evidenced by the upregulation of terminal components; thus, complement inhibition could potentially slow progression.

Objective

To evaluate the safety and efficacy of the terminal complement C5 inhibitor ravulizumab in adults with ALS.

Design, setting, and participants

This double-blind, placebo-controlled, parallel-group, multinational, randomized, phase 3 clinical trial was conducted from March 30, 2020, to October 17, 2021, in 81 ALS specialty centers across 17 countries. A preplanned, unmasked, nonbinding interim futility analysis was conducted when 33% of participants had completed week 26, where

SUBMITTER: Genge A 

PROVIDER: S-EPMC10495927 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature