高用量E0302の筋萎縮性側索硬化症に対する第III相試験

抄録

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects the upper and lower motor neurons. Only riluzole and edaravone are approved as drugs for ALS in the world, and new agents with larger effect size are warranted. Our previous study (E0302–J081–761) has suggested that high–dose mecobalamin (E0302) prolonged the overall survival and suppressed progression in ALS patients with a disease duration less than 12 months in exploratory analyses.

The Japanese Early–stage Trial of high dose methylcobalamin for ALS (JETALS) is a prospective, multicenter, placebo–controlled, double–blind, randomized phase III study conducted at 25 neurology centers and is funded by the Japan Agency for Medical Research and Development.

A total of 128 ALS patients within 1 year from onset were randomized at a 1:1 ratio to receive intramuscular injection with E0302 50mg or placebo twice a week for 16 weeks. The primary endpoint is changes in the ALS Functional Rating Scale Revised (ALSFRS–R) total score at 16 weeks. If the patients expect to receive E0302 50mg after double–blind administration period, E0302 will be provided to those who wish to receive by March 2020 in the continuous administration period.

This study was started in November 2017. The patient enrollment period is scheduled to end on October 2019 and the follow–up is scheduled to end on March 2020. Respectively, 133 and 71 patients were registered for observation and treatment period until Jan, 2019. The number of the patients registered for treatment period has been above the goal.