This randomized double-blind parallel trial (Phase III) was attended by patients aged 20-75 years with grade 1 or 2 ALS from 31 hospitals in Japan. Patients had a score of at least 2 for each of all 12 points of ALSFRS-R, a forced vital lung capacity of ≥ 80% and an established or suspected ALS according to the revised El Escorial criteria. Patients with duration of the disease not exceeding 2 years were subject to inclusion.
Between 28 November 2011 and 03 September 2014, 213 patients were examined and 192 potential participants were selected. Of these, 137 patients completed the full period of follow-up: 69 were randomized to receive edaravone, 68 were randomized to receive placebo. 68 patients who were administered edaravone and 66 who received placebo were included in the primary analysis of efficacy. As a result, the change in ALSFRS-R was -5.01±0.64 in the edaravon group and -7.50±0.66 in the placebo group. The difference between the groups was -2.49 (p=0.0013) in favor of edaravone. 58 (84%) patients treated with edaravone and 57 (84%) patients on placebo reported treatment-related side effects. 11 (16%) patients receiving edaravone and 16 (24%) receiving placebo had serious side effects; 1 (1%) patient receiving edaravone and 4 (6%) patients receiving placebo had adverse events (one case of dysphagia in the edaravone group versus one case of dyspnoea, two cases of respiratory disorders and one case of rash in the placebo group), which resulted in withdrawal of these patients from the study.
Therefore, edaravone has proven its effectiveness in patients with amyotrophic lateral sclerosis (ALS) who met the inclusion criteria, demonstrating a significantly lower rate of reduction in the assessment of ALS progression according to ALSFRS-R versus placebo.