Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients

Edaravone ALS Study Group

Research output: Contribution to journalArticle

106 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)610-617
Number of pages8
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Volume15
Issue number7-8
DOIs
Publication statusPublished - Dec 1 2014

Fingerprint

Amyotrophic Lateral Sclerosis
Placebos
Safety
phenylmethylpyrazolone
Therapeutics
Observation

Keywords

  • ALSFRS-R
  • Amyotrophic lateral sclerosis
  • Edaravone
  • Placebo
  • Randomized trial

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

Cite this

@article{e04042bb738842df98743f372ef2a276,
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.",
keywords = "ALSFRS-R, Amyotrophic lateral sclerosis, Edaravone, Placebo, Randomized trial",
author = "{Edaravone ALS Study Group} and Koji Abe and Yasuto Itoyama and Gen Sobue and Shoji Tsuji and Masashi Aoki and Manabu Doyu and Chikuma Hamada and Kazuoki Kondo and Takatomo Yoneoka and Makoto Akimoto and Hide Yoshino",
year = "2014",
month = "12",
day = "1",
doi = "10.3109/21678421.2014.959024",
language = "English",
volume = "15",
pages = "610--617",
journal = "Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration",
issn = "2167-8421",
publisher = "Informa Healthcare",
number = "7-8",

}

TY - JOUR

T1 - Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients

AU - Edaravone ALS Study Group

AU - Abe, Koji

AU - Itoyama, Yasuto

AU - Sobue, Gen

AU - Tsuji, Shoji

AU - Aoki, Masashi

AU - Doyu, Manabu

AU - Hamada, Chikuma

AU - Kondo, Kazuoki

AU - Yoneoka, Takatomo

AU - Akimoto, Makoto

AU - Yoshino, Hide

PY - 2014/12/1

Y1 - 2014/12/1

N2 - 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.

AB - 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.

KW - ALSFRS-R

KW - Amyotrophic lateral sclerosis

KW - Edaravone

KW - Placebo

KW - Randomized trial

UR - http://www.scopus.com/inward/record.url?scp=84914677322&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84914677322&partnerID=8YFLogxK

U2 - 10.3109/21678421.2014.959024

DO - 10.3109/21678421.2014.959024

M3 - Article

VL - 15

SP - 610

EP - 617

JO - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

JF - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

SN - 2167-8421

IS - 7-8

ER -