Evaluating the safety, tolerability and efficacy of Tirasemtiv in ALS
Research type
Research Study
Full title
A Phase 3, Multi-National, Double-Blind, Randomised, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS)
IRAS ID
183926
Contact name
Catherine Hogben
Contact email
Sponsor organisation
Cytokinetics, Inc.
Eudract number
2014-005413-23
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 6 months, 2 days
Research summary
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that can attack both upper and lower motor neurons and causes degeneration throughout the brain and spinal cord. In patients with ALS, this progressive degeneration eventually leads to the death of the motor neurons.
Tirasemtiv is being investigated as a potential new therapy for the improvement of breathing, muscle weakness and muscle fatigue in patients with ALS. The purpose of this research study is to evaluate the safety and effectiveness of tirasemtiv and how well it is tolerated in patients with ALS. Specifically, this study will evaluate the effects of tirasemtiv on breathing and muscle performance and overall physical function. How much tirasemtiv gets into the blood stream and how long it takes the body to get rid of it will also be measured. Information about any side effects that may occur will also be collected.
Patients with ALS will be assigned to take the study drug twice a day for up to 52 weeks to test its effects on the skeletal muscles involved in breathing and physical function in ALS. It will take approximately 60 weeks to complete this research study. During this time, participants will make 16 study visits to the clinic and undergo various blood tests, clinical assessments and examinations and will be asked to complete participant questionnaires. This is a placebo-controlled study where the eligible participants will have 67% (2 out of 3) chance to receive double-blind tirasemtiv or 33% (1 out of 3) chance to receive placebo (a non-active drug that will look like the investigational study drug). Neither the participant or study doctor will know what the participant is taking in this double-blind study.
Approximately 445 participants with ALS will take part in this research study at approximately 75 hospitals and clinics throughout the United States, Canada and Europe.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
15/EM/0470
Date of REC Opinion
1 Dec 2015
REC opinion
Further Information Favourable Opinion