Long-term edaravone efficacy in amyotrophic lateral sclerosis: Post-hoc analyses of Study 19 (MCI186-19)

Muscle Nerve. 2020 Feb;61(2):218-221. doi: 10.1002/mus.26740. Epub 2019 Nov 11.


Background: In a Phase 3 study, amyotrophic lateral sclerosis (ALS) patients experienced significantly less physical functional decline with 24-week edaravone vs placebo, followed by open-label treatment for an additional 24 weeks.

Methods: Outcome (the change in ALS Functional Rating Scale-Revised, ALSFRS-R, from baseline) was projected for placebo patients through 48 weeks and compared with 48-week edaravone or 24-week edaravone after switching from placebo.

Results: A total of 123 patients received open-label treatment (65 edaravone-edaravone; 58 placebo-edaravone). The projected ALSFRS-R decline for placebo from baseline through week 48 was greater than for 48-week edaravone (P < .0001). For patients switching from placebo to edaravone, ALSFRS-R slope approached that of continued edaravone for 48 weeks. ALSFRS-R decline did not differ between actual and projected edaravone through week 48.

Conclusions: Compared with placebo, these analyses suggest that edaravone is beneficial in ALS patients even after 6 mo of receiving placebo, and efficacy is maintained for up to 1 year.

Keywords: ALSFRS-R; chronic; disease progression; functional decline; linear regression; oxidative stress.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Disease Progression
  • Double-Blind Method
  • Edaravone / therapeutic use*
  • Female
  • Humans
  • Male
  • Middle Aged
  • Neuroprotective Agents / therapeutic use*
  • Treatment Outcome


  • Neuroprotective Agents
  • Edaravone