VITALITY-ALS: Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year

Diagnosis ALS
Study phase Completed
Participating centres UMC Utrecht

A Phase 3, multi-national, double-blind, randomized, placebo-controlled, stratified, parallel group, study to evaluate the safety, tolerability and efficacy of Tirasemtiv in patients with Amyotrophic Lateral Sclerosis (ALS).

This study was to assess the effect of Tirasemtiv versus placebo on respiratory function in patients with ALS.

CY 4031 is a multi-national, double-blind, randomized, placebo-controlled, stratified, parallel group study in patients with ALS with the selective fast skeletal muscle troponin activator, tirasemtiv. The study included three phases; an open-label phase (2 weeks), a double-blind, placebo-controlled phase (48 weeks), and a double-blind, placebo-controlled tirasemtiv withdrawal phase (4 weeks). Patients who completed two weeks of treatment with open-label tirasemtiv (125 mg twice daily) were randomized 3:2:2:2 to placebo and three different dose levels of tirasemtiv. 743 patients were enrolled onto open-label treatment. Patients who entered the study on riluzole 50 mg twice daily continue on riluzole but at a reduced dose of 50 mg once daily.

Performed in more than 75 centers in North-America and Europe.