Data for 'Safety, tolerability and efficacy of the rho kinase inhibitor fasudil in patients with amyotrophic lateral sclerosis (ROCK-ALS): a phase 2, randomised, double-blind, placebo-controlled trial'

This randomised, double-blind, placebo-controlled, phase 2 trial was conducted at 19 ALS centres in Germany, France, and Switzerland. Adult patients with at least probable ALS (revised El Escorial criteria), disease duration of 6-24 months, and a slow vital capacity (SVC) of >65% predicted normal were included. Patients were randomly assigned (1:1:1) to either receive 30 mg (15 mg twice daily) or 60 mg (30 mg twice daily) fasudil or placebo intravenously for 20 days over a 4 week period.. Follow-up assessments were performed at 45, 90 and 180 days after treatment initiation. The co-primary endpoints were safety until day 180 and tolerability during the treatment period. Changes in ALS Functional Rating Scale revised (ALSFRS-R), SVC % predicted, ALS Assessment Questionnaire (ALSAQ-5), Edinburgh Cognitive and Behavioural ALS Screen (ECAS), Motor Unit Number Index (MUNIX), survival, as well as safety during the treatment period were evaluated as secondary endpoints.