A post-hoc subgroup analysis of outcomes in the first phase III clinical study of edaravone (MCI-186) in amyotrophic lateral sclerosis

THE EDARAVONE (MCI-186) ALS 16 STUDY GROUP

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15 Citations (Scopus)

Abstract

Our first phase III study failed to demonstrate efficacy of edaravone for amyotrophic lateral sclerosis (ALS) compared to placebo. Here, we performed post-hoc subgroup analysis to identify a subgroup in which edaravone might be expected to show efficacy. We focussed on two newly defined subgroups, EESP and dpEESP2y. The EESP was defined as the efficacy-expected subpopulation with % forced vital capacity of ≥80%, and ≥2 points for all item scores in the revised ALS functional rating scale (ALSFRS-R) score before treatment. The dpEESP2y was defined as the greater-efficacy-expected subpopulation within EESP having a diagnosis of ‘definite’ or ‘probable’ ALS according to the El Escorial revised Airlie House diagnostic criteria and onset of disease within two years. The primary endpoint of the post-hoc analysis was the change in the ALSFRS-R score during the 24-week treatment period. The intergroup differences of the least-squares mean change in the ALSFRS-R score ± standard error during treatment were 0.65 ± 0.78 (p = 0.4108) in the full analysis set, 2.20 ± 1.03 (p = 0.0360) in the EESP, and 3.01 ± 1.33 (p = 0.0270) in the dpEESP2y. Edaravone exhibited efficacy in the dpEESP2y subgroup. A further clinical study in patients meeting dpEESP2y criteria is warranted.

Original languageEnglish
Pages (from-to)11-19
Number of pages9
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Volume18
DOIs
Publication statusPublished - Oct 31 2017

Keywords

  • Amyotrophic lateral sclerosis (ALS)
  • edaravone
  • MCI-186
  • revised ALS functional rating scale (ALSFRS-R)
  • subgroup analysis

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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