Myoclonus Dystonia Clinical Trial
Official title:
Comparative Study of the Efficiency of Zonisamide in Myoclonus Dystonia: A Monocentric , Randomized in Cross Over and Double Blind Study Versus Placebo Study
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.
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).
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Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment