A Randomized, Double-Blind, Placebo-Controlled Trial Evaluating Cysteamine in Huntington's Disease

作者：Verny Christophe; Bachoud Levi Anne Catherine; Durr Alexandra; Goizet Cyril; Azulay Jean Philippe; Simonin Clemence; Tranchant Christine; Calvas Fabienne; Krystkowiak Pierre; Charles Perrine; Youssov Katia; Scherer Clarisse; Prundean Adriana; Olivier Audrey; Reynier Pascal; Saudou Frederic; Maison Patrick; Allain Philippe; von Studnitz Erica; Bonneau Dominique^*

来源：Movement Disorders, 2017, 32(6): 932-936.

DOI：10.1002/mds.27010

• Huntington's disease
• cysteamine
• clinical trial

Background: Cysteamine has been demonstrated as potentially effective in numerous animal models of Huntington's disease.
Methods: Ninety-six patients with early-stage Huntington's disease were randomized to 1200mg delayed-release cysteamine bitartrate or placebo daily for 18 months. The primary end point was the change from baseline in the UHDRS Total Motor Score. A linear mixed-effects model for repeated measures was used to assess treatment effect, expressed as the least-squares mean difference of cysteamine minus placebo, with negative values indicating less deterioration relative to placebo.
Results: At 18 months, the treatment effect was not statistically significant-least-squares mean difference, -1.5 +/- 1.71 (P=0.385) - although this did represent less mean deterioration from baseline for the treated group relative to placebo. Treatment with cysteamine was safe and well tolerated.
Conclusions: Efficacy of cysteamine was not demonstrated in this study population of patients with Huntington's disease. Post hoc analyses indicate the need for definitive future studies.

• 出版日期2017-6