Randomized phase 2 study of perampanel for sporadic amyotrophic lateral sclerosis

• DOI: 10.1007/s00415-021-10670-y
• Publication Date: 2021-06-30T13:02:56Z
• Authors: Hitoshi Aizawa; Haruhisa Kato; Koji Oba; Takuya Kawahara; Yoshihiko Okubo; Tomoko Saito; Makiko Naito; Makoto Urushitani; Akira Tamaoka; Kiyotaka Nakamagoe; Kazuhiro Ishii; Takashi Kanda; Masahisa Katsuno; Naoki Atsuta; Yasushi Maeda; Makiko Nagai; Kazutoshi Nishiyama; Hiroyuki Ishiura; Tatsushi Toda; Akihiro Kawata; Koji Abe; Ichiro Yabe; Ikuko Takahashi-I...; Hidenao Sasaki; Hitoshi Warita; Masashi Aoki; Gen Sobue; Hidehiro Mizusawa; Yutaka Matsuyama; Tomohiro Haga; Shin Kwak
• Keywords: Perampanel

Abstract Objective To evaluate the efficacy and safety of perampanel in patients with sporadic amyotrophic lateral sclerosis (SALS). Methods This randomized, double-blind, placebo-controlled, multicenter, phase 2 clinical study was conducted at 12 sites. Patients probable or definite ALS as defined by revised El Escorial criteria were enrolled. Sixty-six randomly assigned (1:1:1) to receive placebo, 4 mg perampanel, 8 daily for 48 weeks. Adverse events (AEs) recorded throughout trial period. The primary outcome change Amyotrophic Lateral Sclerosis Rating Scale-Revised (ALSFRS-R) score after weeks treatment. Results One patient withdrew before starting Of 65 included, 18 22 randomized placebo (82%), 14 (64%), 7 21 (33%) completed trial. There a significant difference ALSFRS-R scores [− 8.4 (95% CI − 13.9 2.9); p = 0.015] between group, primarily due worsening bulbar subscore group. Serious AEs more frequent group than ( 0.0483). Conclusions Perampanel associated decline linked