Researchers are looking for people with ALS (amyotrophic lateral sclerosis), to participate in FORTITUDE, a phase 2 clinical trial, sponsored by Cytokinetics, to evaluate the safety, tolerability and effectiveness of the investigational drug CK-2127107.
CK-2127107 is a fast skeletal muscle troponin activator, designed to increase the ability of muscle to contract by sensitizing it to calcium, which it is hoped will lead to increased muscle function and decreased muscle fatigue. In five completed phase 1 trials conducted in healthy volunteers, CK-2127107 proved safe and demonstrated the ability to increase muscle force. Although the approach does not fix the underlying molecular problem in ALS, it could slow the progressive weakness in muscles associated with breathing and voluntary movement in ALS.
There are four treatment groups in the study. Three groups will take varying doses of CK-2127107 and one group will receive placebo. Participants will be randomly assigned to one of the four groups.
CK-2127107 or placebo will be provided as a tablet to be taken twice daily orally.
Trial length will be approximately 18 weeks. This includes screening for up to two weeks, 12 weeks of treatment, and a safety follow-up visit four weeks after the last dose. Following the screening visit, participants will visit the clinic on Day 1, and then on Weeks 2, 4, 8 and 12, and once more for follow-up four weeks after administration of the last dose.
The outcome measures used in this study include slow vital capacity, muscle strength tests, ALS functional rating scale-revised (ALSFRS-R), voice recording, fine motor skills assessment, ALS Assessment Questionnaire (ALSAQ-5) and health economic outcome measures.
In order to be eligible to participate, individuals must be at least 18 years old with a diagnosis of familial or sporadic ALS, be able to swallow tablets and meet additional criteria.
The trial is taking place at study sites at the University of South Florida, Weill Medical College of Cornell University, Ohio State University, Texas Neurology, Stanford University Medical Center, McGill University and West Virginia University Hospitals. Support for travel costs may be available.
To learn more about this trial, visit ClinicalTrials.gov and enter NCT03160898 into the “Other Terms” search box.