A randomized, double-blind, placebo-controlled trial evaluating cysteamine in Huntington's disease

Mov Disord. 2017 Jun;32(6):932-936. doi: 10.1002/mds.27010. Epub 2017 Apr 24.


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 1200 mg 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. © 2017 International Parkinson and Movement Disorder Society.

Keywords: Huntington's disease; clinical trial; cysteamine.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Cysteamine / administration & dosage
  • Cysteamine / adverse effects
  • Cysteamine / pharmacology*
  • Cystine Depleting Agents / administration & dosage
  • Cystine Depleting Agents / adverse effects
  • Cystine Depleting Agents / pharmacology*
  • Delayed-Action Preparations
  • Double-Blind Method
  • Female
  • Follow-Up Studies
  • Humans
  • Huntington Disease / drug therapy*
  • Male
  • Middle Aged
  • Treatment Outcome


  • Cystine Depleting Agents
  • Delayed-Action Preparations
  • Cysteamine