Exploratory double-blind, parallel-group, placebo-controlled study of edaravone(MCI-186) in amyotrophic lateral sclerosis (Japan ALS severity classification: Grade 3, requiring assistance for eating, excretion or ambulation)

Our objective was to explore the efficacy and safety of edaravone in amyotrophic lateral sclerosis (ALS) patients with a Japan ALS severity classification of Grade 3. In a 24 -week, double-blind, randomized study, 25 patients who met all of the follovving criteria were enrolled: Japan ALS severity classification Grade 3; definite, probable, or probable -laboratory supported ALS (El Escorial/revised Airlie House); forced vital capacity (%OFVC)>= 60%; duration of disease <= 3 years at consent; and change in the revised ALS functional rating scale (ALSFRS-R) score of -1 to -4 points during the 12 -week pre -observation period. Patients received edaravone (n=13) or placebo (n=12) for six cycles. The efficacy outcome was change in the ALSFRS-R score. The least -squares mean change in the ALSFRS-R score perpendicular to standard error during the 24 week treatment was -6.52 +/- 1.78 in the edaravone group and -6.00 +/- 1.83 in the placebo group; the difference of -0.52 +/- 2.46 was not statistically significant (p-0.835). Incidence of adverse events was 92.3% (12/13) in the edaravone group and 100.0% (12/12) in the placebo group. There was no intergroup difference in the changes in the ALSFRS-R score. The incidences of adverse events were similar in the two groups.