Cytokinetics Amends Protocol & Revises Guidance For BENEFIT-ALS - Quick Facts

(RTTNews) - Cytokinetics, Inc. (CYTK) announced an amendment to the protocol for BENEFIT-ALS, a Phase IIb, multinational, double-blind, randomized, placebo-controlled study designed to assess the safety, tolerability and potential efficacy of tirasemtiv, a fast skeletal muscle troponin activator, in patients with amyotrophic lateral sclerosis or ALS. The primary analysis of BENEFIT-ALS would compare the mean change from baseline in the ALS Functional Rating Scale in its revised form, or ALSFRS-R, in patients receiving tirasemtiv, compared with those receiving placebo.

Recently, the company said it was informed by its data management vendor that a programming error in the electronic data capture system controlling trial drug assignment caused 58 patients initially randomized to and treated with tirasemtiv to receive placebo instead at a certain study visit and for the remainder of the study. No patients randomized to placebo were dispensed incorrect treatment. Cytokinetics and all trial site personnel remain blinded to the specific patients affected by the error.

Following the error detection, the firm took actions to ensure that no further incorrect study drug assignments occurred and to correct the programming error in the electronic data capture system controlling study drug assignment. Further, it convened an ad hoc meeting of the study's Data Safety Monitoring Board to evaluate if the error in dispensing study drug had impacted the safety of the 58 affected patients.

After interacting with the regulatory authorities, Cytokinetics amended the protocol for BENEFIT-ALS to enable increased enrollment to around 680 patients and to update the statistical methods section, in both cases with the aim of maintaining the originally intended statistical power of the study. To date, more than 500 patients were enrolled in BENEFIT-ALS.

Enrollment is likely to continue as the new protocol amendment becomes effective at participating investigative centers. The company now expects to complete patient enrollment in BENEFIT-ALS in the second half of 2013, with results to be available in early 2014. These changes to BENEFIT-ALS are anticipated to increase the direct trial costs by some $5 million in 2013 and 2014.