title: Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients.
abstract: Our objective was to confirm the efficacy and safety of edaravone in amyotrophic lateral sclerosis (ALS) patients. We conducted a 36-week confirmatory study, consisting of 12-week pre-observation period followed by 24-week treatment period. Patients received placebo or edaravone i.v. infusion over 60 min for the first 14 days in cycle 1, and for 10 of the first 14 days during cycles 2 to 6. The efficacy primary endpoint was changed in the revised ALS functional rating scale (ALSFRS-R) scores during the 24-week treatment. Patients were treated with placebo (n = 104) and edaravone (n = 102). Changes in ALSFRS-R during the 24-week treatment were -6.35 ± 0.84 in the placebo group (n = 99) and -5.70 ± 0.85 in the edaravone group (n = 100), with a difference of 0.65 ± 0.78 (p = 0.411). Adverse events amounted to 88.5% (92/104) in the placebo group and 89.2% (91/102) in the edaravone group. In conclusion, the reduction of ALSFRS-R was smaller in the edaravone group than in the placebo group, but efficacy of edaravone for treatment of ALS was not demonstrated. Levels and frequencies of reported adverse events were similar in the two groups.
authors.affiliation: Department of Neurology, Okayama University Hospital , Okayama.
authors.forename: Koji
authors.initials: K
authors.lastname: Abe
authors.forename: Yasuto
authors.initials: Y
authors.lastname: Itoyama
authors.forename: Gen
authors.initials: G
authors.lastname: Sobue
authors.forename: Shoji
authors.initials: S
authors.lastname: Tsuji
authors.forename: Masashi
authors.initials: M
authors.lastname: Aoki
authors.forename: Manabu
authors.initials: M
authors.lastname: Doyu
authors.forename: Chikuma
authors.initials: C
authors.lastname: Hamada
authors.forename: Kazuoki
authors.initials: K
authors.lastname: Kondo
authors.forename: Takatomo
authors.initials: T
authors.lastname: Yoneoka
authors.forename: Makoto
authors.initials: M
authors.lastname: Akimoto
authors.forename: Hiide
authors.initials: H
authors.lastname: Yoshino
mesh_terms: D000328:Adult; D000368:Aged; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy*; D000704:Analysis of Variance; D000983:Antipyrine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D004311:Double-Blind Method; D000077553:Edaravone; D005260:Female; D005500:Follow-Up Studies; D016166:Free Radical Scavengers / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D012720:Severity of Illness Index; D013997:Time Factors; D055815:Young Adult
chemical_list: D016166:Free Radical Scavengers; D000077553:Edaravone; D000983:Antipyrine
keywords: ALSFRS-R; Amyotrophic lateral sclerosis; edaravone; placebo; randomized trial