Computer Error Results in Wrong Participants Getting Placebo in Tirasemtiv Trial

by Amy Madsen on Mon, 2013-07-08 04:35

58 trial participants mistakenly received a placebo instead of the test drug for part of the BENEFIT-ALS clinical trial of tirasemtiv in ALS

Article Highlights:
  • Cytokinetics' phase 2b clinical trial “BENEFIT-ALS” is designed to evaluate the safety, tolerability and potential efficacy of tirasemtiv in people with amyotrophic lateral sclerosis.
  • Cytokinetics reports that a computer programing error in a data capture system caused 58 people who were randomly assigned to be treated with tirasemtiv to receive a placebo for a portion of the trial; participant safety was not jeopardized.
  • Tirasemtiv is thought to work by increasing the sensitivity of muscle fibers to calcium, resulting in a potential increase in muscle force generation.
  • The trial remains open to enrollment, but Cytokinetics is communicating with regulatory agencies about possible changes in study design.

Update (Dec. 12, 2013): Cytokinetics announced in a Dec. 8, 2013, press release, that the BENEFIT-ALS trial is now fully enrolled and includes 711 participants. Lead investigator Jeremy Shefner presented a discussion of this study at the 24th International Symposium on ALS/MND, held in Milan, Italy. Shefner said the full enrollment at this time may allow results of this trial to be reported during the second quarter of 2014.


Update (July 23, 2013): Cytokinetics announced in a July 23, 2013, press release that it has increased enrollment in the BENEFIT-ALS trial to approximately 680 participants and updated trial statistical methods — allowing the trial to achieve its originally intended statistical power. The company now expects to complete patient enrollment, which currently remains open, in the second half of 2013. Trial results are expected in early 2014.

South San Francisco biotechnology company Cytokinetics announced July 8, 2013, that a computer program error occurred in its phase 2b BENEFIT-ALS clinical trial designed to evaluate the safety, tolerability and potential efficacy of tirasemtiv in people with amyotrophic lateral sclerosis (ALS).

The error caused 58 people who had been randomly assigned to be treated with tirasemtiv to receive a placebo instead for a portion of the trial. No participants who had been assigned to receive placebo received tirasemtiv.

BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv in ALS) is currently designed to enroll up to 500 trial participants; to date, more than 450 people have enrolled.

The trial's data safety monitoring board (DSMB) has reported that safety of the trial participants was not affected by the error in the electronic data capture system, which has since been corrected.

Cytokinetics is seeking regulatory guidance

Cytokinetics may make changes in the trial, pending discussion with regulatory authorities.

"Cytokinetics is in communication with regulatory authorities regarding how best to respond to the error in drug assignment in order to preserve the intended scientific value of BENEFIT-ALS," the company said in a July 8, 2013, press release. "The company continues to enroll patients in the study under the current protocol and may amend the protocol to allow increased enrollment."

The release went on to say, "Following further communications with regulatory authorities, Cytokinetics expects to provide updated guidance relating to the conduct of BENEFIT-ALS, which may include revisions to the timing of publicly available results from the study as well as to the projected costs of the study."

For more information about BENEFIT-ALS, see Study of Safety, Tolerability and Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS) or enter NCT01709149 in the search box at ClinicalTrials.gov.

More about tirasemtiv

Tirasemtiv (formerly CK-2017357) is thought to work by increasing the sensitivity of muscle fibers to calcium, resulting in a potential increase in muscle force generation. (To read a manuscript describing tirasemtiv's mechanism of action, visit the Cytokinetics home page and select the Nature Medicine paper on activation of fast skeletal muscle troponin under "Recent Scientific Publications.")

Data from two completed phase 2 clinical trials of tirasemtiv in ALS were presented at the April 2012 American Academy of Neurology (AAN) Annual Meeting in New Orleans. In both trials, the experimental drug was generally safe and well-tolerated.

The U.S. Food and Drug Administration (FDA) granted tirasemtiv orphan drug status March 10, 2010. (Orphan drug status provides financial incentives for the development of drugs for rare diseases.)

Cytokinetics also is testing tirasemtiv in myasthenia gravis (MG); the company expanded development and testing of the drug in April 2013 to include spinal muscular atrophy (SMA).

About Clinical Trials

About Clinical Trials

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 Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to ClinicalTrials.gov.

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