Investigators are seeking participants for an ongoing phase 2 clinical trial, sponsored by Cytokinetics, to test the investigational drug CK-2127107 in people with types 2, 3 or 4 spinal muscular atrophy (SMA).
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 SMA, drugs that enhance muscle function could potentially be used in combination with other therapies that act on the genetic cause of the disease.
Following a review of safety and other data from the first group of participants in this trial, investigators now are seeking additional participants in which to test a higher dose of the drug. At seven visits to the trial site over a period of 12 weeks, trial participants will undergo multiple assessments of skeletal muscle function and fatigability including respiratory tests, upper limb strength and functionality assessments for non-ambulatory patients, and a six-minute walk and timed-up-and-go test for ambulatory patients.
Trial investigators aim to evaluate safety and tolerability of CK-2127107 and determine whether the drug has beneficial effects in SMA.
Study participants must be 12 years or older and meet additional eligibility criteria. Support for travel costs may be available.
To learn more about this trial, including inclusion and exclusion criteria and trial site locations, visit ClinicalTrials.gov and enter NCT02644668 into the search box.