Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.

ABSTRACT:

Importance

Amyotrophic lateral sclerosis (ALS) is a fatal disease. The sigma-1 (σ1) receptor emerged as a target for intervention.

Objective

To determine the effects of pridopidine, a σ1-receptor agonist, in ALS.

Design, settings, and participants

Pridopidine was tested as a regimen of the HEALEY ALS Platform Trial, a phase 2/3, multicenter, randomized, double-blind, platform trial. The study was conducted at 54 sites in the US from January 2021 to July 2022 (final follow-up, July 14, 2022). A total of 163 participants with ALS were randomized to receive pridopidine or placebo. An additional 122 concurrently randomized participants were assigned to receive placebo in other regimens and included in the analyses.

Interventions

Eligible participants were randomize

SUBMITTER: Writing Committee for the HEALEY ALS Platform Trial 

PROVIDER: S-EPMC11833658 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature