A controlled trial of
idebenone in Huntington's disease.
Ranen NG, Peyser CE, Coyle JT, Bylsma FW,
Sherr M, Day L, Folstein MF, Brandt
Department of Psychiatry,
Johns Hopkins University School of Medicine,
Baltimore, Maryland, USA.
Mov Disord 1996 Sep;11(5):549-54
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.