Ethyl-EPA in Huntington disease: A double-blind, randomized, placebo-controlled trial

B. K. Puri, B. R. Leavitt, M. R. Hayden, C. A. Ross, A. Rosenblatt, J. T. Greenamyre, S. Hersch, K. S. Vaddadi, A. Sword, D. F. Horrobin, M. Manku, Harald Murck

Research output: Contribution to journalArticlepeer-review


Background: Preliminary evidence suggests beneficial effects of pure ethyl-eicosapentaenoate (ethyl-EPA) in Huntington disease (HD). Methods: A total of 135 patients with HD were randomized to enter a multicenter. double-blind, placebo-controlled trial on the efficacy of 2 g/d ethyl-EPA vs placebo. The Unified Huntington's Disease Rating Scale (UHDRS) was used for assessment. The primary end point was outcome at 12 months on the Total Motor Score 4 subscale (TMS-4). Analysis of covariance (ANCOVA) and a χ2 test on response, defined as absence of increase in the TMS-4, were performed. Results: A total of 121 patients completed 12 months, and 83 did so without protocol violations (PP cohort). Intent-to-treat (ITT) analysis revealed no significant difference between ethyl-EPA and placebo for TMS-4. In the PP cohort, ethyl-EPA proved better than placebo on the χ2 test on TMS-4 (p < 0.05), but missed significance on ANCOVA (p = 0.06). Secondary end points (ITT cohort) showed no benefit of ethyl-EPA but a significantly worse outcome in the behavioral severity and frequency compared with placebo. Exploring moderators of the efficacy of ethyl-EPA on TMS-4 showed a significant interaction between treatment and a factor defining patients with high vs low CAG repeats. Reported adverse events were distributed equally between treatment arms. Conclusions: Ethyl-eicosapentaenoate (ethyl-EPA) (purity >95%) had no benefit in the intent-to-treat cohort of patients with Huntington disease, but exploratory analysis revealed that a significantly higher number of patients in the per protocol cohort, treated with ethyl-EPA, showed stable or improved motor function. Further studies of the potential efficacy of ethyl-EPA are warranted.

Original languageEnglish (US)
Pages (from-to)286-292
Number of pages7
Issue number2
StatePublished - Jul 26 2005

ASJC Scopus subject areas

  • Clinical Neurology


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