Efficacy Trial of Zonisamide for Myoclonus Dystonia
Condition: Myoclonus Dystonia
MeSH terms: Dystonia; Dystonic Disorders; Myoclonus
Study type: Interventional
Phase: Phase 3

Summary: 
      Myoclonus Dystonia is a disease in which myoclonus distort the precision of movements and so
      cause a handicap in the movements of the everyday life. Response to oral medications may be
      incomplete and surgery may cause operating risk.

      Zonisamide is an antiepileptic drug which could bring a therapeutic profit in Myoclonus
      Dystonia on the severity of the myoclonus.
    

Detailed description: 
      In "dystonia", the involuntary abnormal movements cause a driving handicap and a change of
      the quality of life. A particular shape of dystonia, the Myoclonus Dystonia, is characterized
      by the ascendancy of myoclonias (abrupt and brief movements) associated with the abnormal
      dystonia. Myoclonus is an additional source of handicap in the movements of the everyday
      life, because they distort the precision of movements. Response to oral medications may be
      incomplete and the tolerance poor, such that deep brain stimulation (DBS) surgery is useful
      for the major forms but it is also an invasive therapeutics which the operating risk is not
      totally estimated in the absence of controlled study. Therefore, it is necessary to
      investigate other pharmacological therapeutic tracks which present a good ratio profit /
      risk.

      Zonisamide is usually used in France in the epilepsy's treatment. It showed its efficiency in
      the progressive myoclonus epilepsy, not only on the seizure but also on the myoclonia.
      Therefore, it showed its efficiency on post-anoxic and propriospinal myoclonus. So, we make
      the hypothesis that this medicine could bring a therapeutic profit in the Myoclonus Dystonia.

      The aim of this study is to demonstrate the efficiency of the zonisamide on the severity of
      myoclonus (UMRS) at those patients. The others outcomes are to estimate the impact of the
      treatment on the myoclonus's neurophysiological characteristics, the dystonia's severity (BFM
      score), the quality of life (SF-36 and CGI scores), but also to investigate the tolerance of
      the treatment.

      We conducted a randomized, placebo-controlled, double-blind, two-period cross-over design to
      evaluate the effect on severity of myoclonus in response to placebo or zonisamide (until 300
      mg) in 32 patients.

      The study includes an evaluation at the beginning and at the end of every period (4
      evaluations at all). Each period includes a phase of titration (six weeks) followed by a
      phase of fixed dose (three weeks). Those two periods are separated by a period of wash-out (3
      weeks) preceded by a phase of progressive decrease of doses (two weeks).
    

Intervention name(s): zonegran; placebo
Intervention type(s): Drug; Drug
Intervention MeSH terms: Zonisamide
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Movement disorder; Myoclonus dystonia; DYT 11; Antiepileptic drugs; Zonisamide; Clinical Trial; Double-mind method