Investigator–initiated clinical trial for amyotrophic lateral sclerosis

Abstract

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. Recently, investigator–initiated clinical trial of ALS with Hepatocyte Growth Factor, Perampanel, high dose of methylcobalamin, Ropinirole, and Bosutinib were conducted in Japan.

The Japan 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. At JETALS, we will evaluate the efficacy and safety of high dose methylcobalamin, evaluate that the updated Awaji criteria are useful for early diagnosis of ALS, and verify that high dose methylcobalamin can be appropriately administered by self–administration.

JETLAS 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 2022.