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 (VITALITY-ALS)
Study Purpose:
This study is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
Active, no longer recruitingPhase:
Phase IIIStudy Chair(s)/Principal Investigator(s):
Study Director: MD Cytokinetics
Clinicaltrials.gov ID (11 digit #):
NCT02496767Neals Affiliated?
YesCoordinating Center Contact Information
MD CytokineticsSarah Kulke, MD / .(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) United States
Full Study Summary:
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 includes 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 can complete two weeks of treatment with open-label tirasemtiv (125 mg twice daily) will be randomized 3:2:2:2 to placebo and three different dose levels of tirasemtiv. Approximately 445 patients will be enrolled onto open-label treatment. Patients who enter the study on riluzole 50 mg twice daily will continue on riluzole but at a reduced dose of 50 mg once daily.
Primary Outcome Measures:
-Change from baseline to Week 24 of the double-blind, placebo-controlled phase in percent predicted slow vital capacity (SVC) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
-Time to the first occurrence of a decline in the respiratory components of the ALS functional rating scale- revised (ALSFRS-R) (i.e., items 10, 11, and 12) or death during double-blind, placebo-controlled treatment [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
-Time to the first occurrence of a decline from baseline in percent predicted SVC ≥ 20 percentage points or the onset of respiratory insufficiency or death during double-blind, placebo-controlled treatment [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
-Time to the first occurrence of any use of assisted ventilation or death during double-blind, placebo-controlled treatment [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
-Time to the first occurrence of a decline in SVC to ≤ 50% predicted or the onset of respiratory insufficiency or death during double-blind, placebo-controlled treatment [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
-Slope in muscle strength mega-score change from baseline during the randomized, double-blind, placebo-controlled phase [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]