Largest HD Clinical Trial Halted

Coenzyme Q10 “very unlikely” to show significant benefit in HD in 2CARE study

The largest randomized, controlled trial ever conducted in HD has been halted for futility. On August 9, 2014, the Huntington Study Group (HSG) reported that the ongoing 2CARE study, a phase 3 trial of Coenzyme Q10 (CoQ10) in HD (see HD Insights ,Vol. 6), was stopped by the National Institutes of Neurological Disorders and Stroke on July 14, 2014, after an interim analysis conducted by the Data and Safety Monitoring Board found that it would be “very unlikely” to see a significant effect of 2400 mg CoQ10 compared to placebo by the scheduled end of the trial.1 The results, while disappointing, are consistent with prior trials of CoQ10 for neurological disorders such as Parkinson disease, in which the QE3 phase III trial of CoQ10 was also halted prematurely for futility.2

The 2CARE study was a randomized, double-blind, placebo-controlled study conducted by the HSG under the direction of Merit Cudkowicz, MD, MSc, from Massachusetts General Hospital, Michael McDermott, PhD, from the Biostatistics department at the University of Rochester, and Karl Kieburtz, MD, MPH, Director of the University of Rochester’s Clinical and Translational Sciences Institute. Beginning in March 2008, the trial enrolled 609 individuals with HD in 48 sites in Australia and North America. Data collection was scheduled to end in August 2017. Despite the failure of CoQ10, the study’s leaders report that the data collected in the study will certainly prove useful in future research. Dr. Merit Cudkowicz commented that, “We will make this dataset available to all HD researchers… to mine for new ideas about HD. [This] is the longest follow up dataset from any clinical trial and should lead to new insights and directions.” Dr. Karl Kieburtz agreed, saying, “While CoQ[10] does not appear useful in HD, the work itself is not ‘futile’ since we expect to have learned a great deal about HD progression.”