Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS.

ABSTRACT: OBJECTIVE:To examine the safety and tolerability as well as the preliminary efficacy of arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in patients with rapidly progressive SOD1 amyotrophic lateral sclerosis (ALS). METHODS:This was a double-blind, placebo-controlled trial in which patients with rapidly progressive SOD1-mutant ALS were randomized 1:1 to receive arimoclomol 200 mg tid or matching placebo for up to 12 months. Study procedures were performed using a mix of in-person and remote assessments. Primary outcome was safety and tolerability. Secondary outcome was efficacy, with survival as the principal measure. Additional efficacy measures were the rates of decline of the Revised ALS Functional Rating Scale (ALSFRS-R) and percent predicted forced e

SUBMITTER: Benatar M 

PROVIDER: S-EPMC5818014 | biostudies-literature | 2018 Feb

REPOSITORIES: biostudies-literature