Investigators are seeking participants for a new 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. In five completed phase 1 trials conducted in healthy volunteers, CK-2127107 proved safe and able to increase muscle force. Although the approach does not fix the underlying molecular problem in SMA, drugs that enhance muscle function could likely be used in combination with other therapies that act on the genetic cause of the disease.

Investigators plan to enroll 72 patients in the trial; each will randomly be assigned treatment over a period of eight weeks with CK-2127107 or a placebo. Multiple assessments of skeletal muscle function and fatigability will be performed including respiratory assessments, upper limb strength and functionality for non-ambulatory patients, as well as six-minute walk and timed-up-and-go for ambulatory patients.

The new study aims to evaluate safety and tolerability and determine whether CK-2127107 has beneficial effects in SMA.

Study participants must be 12 years or older and meet other eligibility criteria.

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.

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