The main efficacy of edaravone injection is to treat amyotrophic lateral sclerosis (ALS). The following describes the efficacy of edaravone in the treatment of ALS in conjunction with clinical trials.
Efficacy of edaravone in the treatment of ALS
Clinical trial inclusion criteria
The efficacy of edaravone in the treatment of ALS was determined in a 6-month randomized, placebo-controlled, double-blind study in Japanese ALS patients who were independently living and met the following criteria at screening: patients retained most activities of daily living (defined as a score of 2 or more on each item of the ALS Functional Rating Scale); normal respiratory function (percent predicted forced vital capacity [%FVC] ≥ 80%); confirmed or suspected ALS according to the El Escorial revised criteria; disease duration of 2 years or less.
Trial design
The study enrolled 69 patients treated with edaravone and 68 patients treated with placebo. The baseline characteristics of the two groups were similar, and more than 90% of the patients in each group were treated with riluzole. Edaravone was administered as an intravenous infusion of 60 mg over 60 minutes according to the following schedule: initial treatment cycle of 14 days, daily dosing, followed by a 14-day drug-free period (Cycle 1); subsequent treatment cycles were 14 days, daily dosing for 10 days, followed by a 14-day drug-free period (Cycles 2-6).
Trial Results
The primary efficacy endpoint was to compare the change in ALSFRS-R total score from baseline to Week 24 between treatment groups. The ALSFRS-R scale consists of 12 questions that assess fine motor, gross motor, bulbar, and respiratory function (speech, salivation, swallowing, writing, cutting food, dressing/hygiene, turning over in bed, walking, climbing stairs, dyspnea, direct breathing, and respiratory insufficiency) in patients with ALS. Scores for each item range from 0 to 4, with higher scores representing greater functional ability.
Compared with the placebo group, the ALSFRS-R score of patients in the edaravone group decreased much less from baseline, with the score in the edaravone group decreasing by 5.01±0.64 from baseline and the score in the placebo group decreasing by 7.50±0.66 from baseline, with a treatment difference of 2.49 (0.99, 3.98). The conclusion was drawn by analyzing the percentage distribution of the change in ALSFRS-R score from baseline to week 24.
In summary, edaravone can significantly delay the onset of amyotrophic lateral sclerosis.
