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A randomized, controlled, double-blind, crossover trial of zonisamide in myoclonus-dystonia
Elodie Hainque1, Marie Vidailhet2, Nathalie Cozic1
1From Université de la Sorbonne UPMC Paris 06 UMR S 1127 (E.H., M.V., V.B., C.H., D.G., A. Méneret, S. Dupont, E.A., J.-C.C., E.R.), Inserm U 1127, CIC-1422, CNRS UMR 7225, Institut du Cerveau et de la Moëlle, Paris; Département des Maladies du Système Nerveux (E.H., M.V., C.H., B.D., C.B., D.G., J.-C.C., E.R.), Département de Biostatistiques, Unité de Recherche Clinique (N.C., A.B., A. Mallet), Pharmacie (F.C.-B.), Département de Pharmacologie (N.Z.), Département de Génétique, UF de Neurogénétique Moléculaire et Cellulaire (F.C.), and Département d'Epilepsie et de Réhabilitation (S. Dupont), Hôpital Pitié-Salpêtrière, AP-HP; Unité de Neurophysiologie (E.H., E.A.), Département DéPAS, Hôpital Saint-Antoine, AP-HP, Paris; Hospices Civils de Lyon (S.T.), Hôpital Neurologique Pierre Wertheimer; Université Lyon 1 (S.T.); CNRS (S.T.), Centre de Neurosciences Cognitives, UMR 5229, Bron; Département de Neurologie (C.T.), Hôpital de Hautepierre, CHU Strasbourg; Fédération de Médecine translationnelle de Strasbourg (FMTS) (C.T.), Strasbourg, France; Département de Neurologie (G.G.), Hôpital Brugmann, Bruxelles, Belgium; Département de Neurologie (S.Drapier), Hôpital Pontchaillou, CHU Rennes; EA-4712 "Comportement et Noyaux Gris Centraux" (S. Drapier), Université de Rennes 1; Département de Neurologie et Pathologies du Movement (E.M.) and Département de Neurologie (T.L.), CHU de Lille; INSERM UMR-S 1172 (E.M.), Lille; Département de Neurologie (A.D.D.M.), Hôpital Maison Blanche, CHU de Reims; Centre Memoire de Ressources et de Recherche (CMRR) (T.L.), Lille; ESPCI Paris Tech (A.-P.L.), PSL Research University; and Département de Neurologie et Pathologie du Movement (J.-P.A.), Pôle Neurosciences Cliniques, INT-CNRS/AMU Aix-Marseille, France.
Objective:
To evaluate the efficacy and safety of zonisamide in patients with myoclonus-dystonia.
Methods:
We conducted a randomized, double-blind, placebo-controlled crossover trial of zonisamide (300 mg/d) in 24 patients with myoclonus-dystonia. Each treatment period consisted of a 6-week titration phase followed by a 3-week fixed-dose phase. The periods were separated by a 5-week washout period. The co-primary outcomes were action myoclonus severity (section 4 of the Unified Myoclonus Rating Scale [UMRS 4]) and myoclonus-related functional disability (UMRS 5). Secondary outcomes included dystonia severity, assessed with the movement and disability subscales of the Burke-Fahn-Marsden-Dystonia Rating Scale (BFM), the Clinical Global Impression-Improvement scale (CGI), and safety measures. Wilcoxon signed-rank tests for paired data were used to analyze treatment effects.
Results:
Twenty-three patients (11 men, 12 women) were analyzed in the intention-to-treat analysis. Zonisamide significantly improved both action myoclonus (median improvement [95% confidence limits] -5 [-9.25 to -1.44], p = 0.003) and myoclonus-related functional disability (median improvement [95% confidence limits] -2 [-2.58 to -2.46], p = 0.007) compared to placebo. Zonisamide also significantly improved dystonia (BFM movement) compared to placebo (median improvement [95% confidence limits] -3 [-8.46 to 0.03], p = 0.009). No difference was found between zonisamide and placebo with respect to the CGI (median improvement [95% confidence limits] -1 [-1.31 to 0.09], p = 0.1). Zonisamide was well-tolerated.
Conclusions:
Zonisamide is well-tolerated and effective on the motor symptoms of myoclonus-dystonia.
Classification Of Evidence:
This study provides Class I evidence that zonisamide improves myoclonus and related disability in patients with myoclonus-dystonia.
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