Dosage effects of riluzole in Huntington's disease: a multicenter placebo-controlled study

Neurology. 2003 Dec 9;61(11):1551-6. doi: 10.1212/01.wnl.0000096019.71649.2b.


Background: Riluzole retards striatal glutamate release and pathologic consequences in neurotoxic animal models of Huntington's disease (HD).

Objective: To determine the dosage-related impact of riluzole on chorea in HD.

Methods: An 8-week double-blind dose-ranging multicenter study of riluzole was conducted in 63 subjects (32 women, 31 men) with HD who were randomized to receive placebo, riluzole 100 mg/day, or riluzole 200 mg/day. The prespecified outcome measure was change in the total maximal chorea score of the Unified Huntington's Disease Rating Scale (UHDRS).

Results: Fifty-six (89%) subjects completed the study. A reduction (p < 0.01) in chorea at 8 weeks was found using a linear trend test with dose. Comparing the groups individually, the reduction in chorea for the riluzole 200-mg/day group (-2.2 +/- 3.3) was different (p = 0.01) from placebo (+0.7 +/- 3.4), but the riluzole 100-mg/day group (-0.2 +/- 2.9) was not. Riluzole did not improve other motor, cognitive, behavioral, or functional components of the UHDRS. Alanine aminotransferase was elevated in a dosage-dependent fashion (p = 0.01).

Conclusions: Over 8 weeks of treatment, riluzole 200 mg/day ameliorated chorea intensity in HD without improving functional capacity or other clinical features of illness. Riluzole 200 mg/day was attended by reversible liver transaminase abnormalities that would require monitoring in long-term studies.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Chorea / drug therapy
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Excitatory Amino Acid Antagonists / administration & dosage*
  • Excitatory Amino Acid Antagonists / adverse effects
  • Excitatory Amino Acid Antagonists / therapeutic use
  • Female
  • Humans
  • Huntington Disease / diagnosis
  • Huntington Disease / drug therapy*
  • Male
  • Middle Aged
  • Riluzole / administration & dosage*
  • Riluzole / adverse effects
  • Riluzole / therapeutic use
  • Treatment Outcome


  • Excitatory Amino Acid Antagonists
  • Riluzole