- A second phase 2 clinical trial of the experimental ALS treatment CK-2017357 has launched.
- Researchers expect to enroll approximately 24 people with ALS for the study, which will be conducted at eight to 10 centers in the United States.
- The trial's primary goal is to assess the safety and tolerability of CK-2017357 in people with ALS; secondary outcome measures include evaluation of participants' motor function, pulmonary (breathing) function, and muscle strength and fatigue.
San Francisco biotechnology company Cytokinetics announced June 21, 2011, that it has opened enrollment for a new clinical trial of CK-2017357 at three different dosage levels in people with ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).
The new trial marks the second phase 2 study for CK-2017357, Cytokinetics' lead drug candidate for ALS. The first phase 2 trial of CK-2017357, which has been completed, had a different study design from the new trial.
CK-2017357 is thought to increase the sensitivity of muscle fibers to calcium, resulting in a potential increase in muscle force generation.
It was granted orphan drug status March 10, 2010, by the U.S. Food and Drug Administration (FDA). (Orphan drug status provides financial incentives for the development of drugs for rare diseases.)
New trial will look at safety, benefit
"This clinical trial is the seventh in a series of trials designed to evaluate CK-2017357 for the potential treatment of patients with muscle dysfunction or impairment [in ALS and other diseases]," said Cytokinetics' president and CEO Robert Blum in a press release posted June 21, 2011, on the company's website. He noted that the development of CK-2017357 for ALS is a priority for Cytokinetics.
The new trial is designed to assess the safety and tolerability of CK-2017357 in people with ALS who will take a series of oral doses of the drug for two weeks.
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur. MDA has no ability to influence who is chosen to participate in a clinical trial. To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to www.clinicaltrials.gov.
Researchers also will look at secondary outcome measures, including patients' motor function (as measured using the ALS Functional Rating Scale-revised, or ALSFRS-R), pulmonary function, muscle strength and fatigue.
Trial participants will be randomly assigned to one of four groups in which they will receive oral CK-2017357 at a dose of 125, 250 or 375 milligrams per day or a placebo during a two-week period. Neither the participants nor the investigators will know who is taking a placebo or who is receiving a particular dosage level of the experimental drug.
Clinical assessments will be made during the two-week treatment phase, and again one week after treatment ends.
Encouraging results were obtained in an earlier phase 2 trial
Jeremy Shefner, professor and chair of neurology at Upstate Medical University, State University of New York in Syracuse, reported favorable results from Cytokinetics' earlier phase 2 ALS clinical trial of CK-2017357 on Dec. 13, 2010, at the 21st International Symposium on ALS/MND in Orlando, Fla.
The drug proved to be safe and well-tolerated in people with ALS, and participants showed statistically significant improvements in breathing ability and muscle strength.
Shefner directs the MDA/ALS Center at his institution, but MDA is not involved with this study.
For information on study center locations and eligibility requirements for participation in this trial, see Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients with ALS. (You can search for this trial on ClinicalTrials.gov under trial ID NCT01378676.)
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