A randomized, placebo-controlled trial of AFQ056 for the treatment of chorea in Huntington's disease

Ralf Reilmann, Marie-Laure Rouzade-Dominguez, Carsten Saft, Sigurd D Süssmuth, Josef Priller, Anne Rosser, Hugh Rickards, Ludger Schöls, Nicole Pezous, Fabrizio Gasparini, Donald Johns, Georg Bernhard Landwehrmeyer, Baltazar Gomez-Mancilla

Research output: Contribution to journalArticlepeer-review

Abstract / Description of output

BACKGROUND: This study investigated the hypothesis that AFQ056 (mavoglurant), a selective metabotropic glutamate receptor 5 antagonist, reduces chorea in Huntington's disease (HD).

METHODS: This 32-day randomized, double-blind, parallel-group, proof-of-concept study investigated AFQ056 (25-150 mg [incremental doses], twice-daily) versus placebo in patients with HD. Primary efficacy assessments were the chorea-sum score and orientation index (nondominant hand) from the quantitative motor (Q-Motor) grasping task at day 28. Key secondary efficacy assessments included finger-tapping in the Unified Huntington's Disease Rating Scale-Total Motor Score and Q-Motor measures. Safety and tolerability were assessed.

RESULTS: Overall, 42 patients were randomized. At day 28, no improvement was observed on the primary efficacy assessments (P > 0.10) with AFQ056 versus placebo. The Q-Motor speeded-tapping interonset interval variability was reduced with AFQ056 versus placebo for the nondominant hand (P = 0.01). The incidence of adverse events was 66.7% with AFQ056 and 57.1% with placebo.

CONCLUSIONS: AFQ056 did not reduce choreatic movements in HD, but was well tolerated. The clinical relevance of the Q-Motor findings (speeded-tapping) are unknown and may warrant further investigation.

Original languageEnglish
Pages (from-to)427-31
Number of pages5
JournalMovement Disorders
Issue number3
Early online date17 Feb 2015
Publication statusE-pub ahead of print - 17 Feb 2015

Keywords / Materials (for Non-textual outputs)

  • Adult
  • Aged
  • Aged, 80 and over
  • Analysis of Variance
  • Chorea
  • Disability Evaluation
  • Double-Blind Method
  • Excitatory Amino Acid Antagonists
  • Female
  • Humans
  • Huntington Disease
  • Indoles
  • Male
  • Middle Aged
  • Severity of Illness Index
  • Time Factors
  • Treatment Outcome
  • Journal Article
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't


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