A controlled trial of idebenone in Huntington's disease.

Ranen NG, Peyser CE, Coyle JT, Bylsma FW, 
Sherr M, Day L, Folstein MF, Brandt J

Department of Psychiatry, 
Johns Hopkins University School of Medicine, 
Baltimore, Maryland, USA.
Mov Disord 1996 Sep;11(5):549-54

ABSTRACT

One hundred patients with clinically diagnosed Huntington's disease (HD) were randomized to either idebenone, an antioxidant and enhancer of oxidative metabolism, or placebo, in a 1-year, double-blind, parallel-group study aimed at slowing the rate of progression of the disease. Ninety-one patients completed the study. There were no significant differences between groups on the primary outcome measures of the Huntington's Disease Activities of Daily Living Scale (ADL-an index of functional status) and the Quantified Neurologic Examination (QNE). Sample size calculations based on progression of the ADL and QNE in this study group revealed that a larger study group is necessary to detect any differences less than an almost complete halting of the disease. This argues for multicenter efforts for future therapeutic trials in HD.