Data Management Vendor’s Error Causes Cytokinetics to Revaluate ALS Trial

08-Jul-2013 - Last updated on 09-Jul-2013 at 12:31 GMT

Related tags Pharmacology Food and drug administration

Data Management Vendor’s Error Causes Cytokinetics to Revaluate Trial

Programming errors in a vendor's EDC system meant some patients were mistakenly given placebo during a trial of an ALS (amyotrophic lateral sclerosis) drug candidate being developed by Cytokinetics.

San Francisco, US-based Cytokinetics said that as a result of the error, 58 patients in a Phase IIb study who had initially been randomized to receive the drug candidate tirasemtiv had been given placebo.

The developer explained that it had been notified of the problem by the electronic data capture (EDC) vendor - which it did not name, but which the San Francisco Business Times said is a CRO - and stressed that subsequent analysis by the study data safety monitoring board revealed that the error did not impact patient safety.

It also said that it is working to enroll 50 more patients and may amend the protocol to increase enrollment.

Discussions with regulatory authorities will center on how to deal with the error and preserve the scientific value of the trial, Cytokinetics said. Updates following these discussions might include revisions to the study’s cost or changes to the results timetable. The firm previously said the study should be complete by about October, according to the US NIH’s ClinicalTrials.gov.

Since correcting the programming error, the company said it is further ensuring that no incorrect drug assignments have occurred in the study. Cytokinetics did not respond to a request for comment on what type of EDC system caused the error.

The treatment involved in the study - tirasemtiv, which is a fast skeletal muscle troponin activator - was granted orphan drug designation and fast track status by the US FDA, as well as an orphan medicinal product designation by the EMA (European Medicines Agency) for the treatment of ALS.

Cytokinetics is also collaborating with Astellas Pharma to develop a similar investigative treatment for non-neuromuscular indications.

Shares of the company’s stock were down almost 9% early Monday but then rebounded to finish the day up almost 5%. Jeremy Shefner, MD, PhD of the State University of New York - Upstate Medical University is the study’s chair and principal investigator.