Dorsey ER, Shoulson I, Leavitt B, Ross C, Beck CA, de Blieck EA, Greenamyre JT, Hersch SM, Kieburtz K, Marder K, McCallum C, Moskowitz C, Oakes D, Rosenblatt A, Shinaman A, Frucht S, Margolis R, Corey-Bloom J, Hersch SM, Mook L, Shannon K, Jaglin J, Sanchez-Ramos J, Wheelock V, Tempkin T, Dure LS, Guttman M, Feigin A, Shannon B, Anderson KE, Racette BA, Higgins D, Agarwal P, Seeberger L, Montellano S, Kostyk S, Seward A, Nance M, Raymond LA, Decolongon J, Suchowersky O, Beglinger L, Paulson H, Como P, Barbano R, Zimmerman C, Wojcieszek J, Jog M, Horn C, Dubinsky RM, Martin W, Wieler M, LeDoux MS, Harrison MB, Morgan JC, Dill B, Singer C, Quesada M, Kartha N, Wernette K, Frank S, Fernandez H, Jennings D, Kelsey T, Hunter C, Beck C, Bourgeois K, de Blieck EA, Deuel L, McCallum C, McMullen N, Oakes D, Ross V, Rumfola L, Watts A, Weaver C, Winebrenner T, Tariot PN.
Abstract
OBJECTIVE:
To determine whether ethyl-eicosapentaenoic acid (ethyl-EPA), an omega-3 fatty acid, improves the motor features of Huntington disease.
DESIGN:
Six-month multicenter, randomized, double-blind, placebo-controlled trial followed by a 6-month open-label phase without disclosing initial treatment assignments.
SETTING:
Forty-one research sites in the United States and Canada.
PATIENTS:
Three hundred sixteen adults with Huntington disease, enriched for a population with shorter trinucleotide (cytosine-adenine-guanine) repeat length expansions.
INTERVENTIONS:
Random assignment to placebo or ethyl-EPA, 1 g twice a day, followed by open-label treatment with ethyl-EPA.
MAIN OUTCOME MEASURES:
Six-month change in the Total Motor Score 4 component of the Unified Huntington's Disease Rating Scale analyzed for all research participants and those with shorter cytosine-adenine-guanine repeat length expansions (<45).
RESULTS:
At 6 months, the Total Motor Score 4 point change for patients receiving ethyl-EPA did not differ from that for those receiving placebo. No differences were found in measures of function, cognition, or global impression. Before public disclosure of the 6-month placebo-controlled results, 192 individuals completed the open-label phase. The Total Motor Score 4 change did not worsen for those who received active treatment for 12 continuous months compared with those who received active treatment for only 6 months (2.0-point worsening; P=.02).
CONCLUSION:
Ethyl-EPA was not beneficial in patients with Huntington disease during 6 months of placebo-controlled evaluation. Clinical Trial Registry clinicaltrials.gov Identifier: NCT00146211.