A randomized, double-blind, placebo-controlled trial evaluating cysteamine in Huntington’s disease.

Christophe Verny, Anne-Catherine Bachoud-Lévi, Alexandra Durr, Cyril Goizet, Jean-Philippe Azulay, Clémence Simonin, Christine Tranchant, Fabienne Calvas, Pierre Krystkowiak, Perrine Charles, Katia Youssov, Clarisse Scherer, Adriana Prundean, Audrey Olivier, Pascal Reynier, Frédéric Saudou, Patrick Maison, Philippe Allain, Erica von Studnitz, Dominique Bonneau,
Mov Disord.. 2017-04-24; 32(6): 932-936
DOI: 10.1002/mds.27010

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1. Mov Disord. 2017 Jun;32(6):932-936. doi: 10.1002/mds.27010. Epub 2017 Apr 24.

A randomized, double-blind, placebo-controlled trial evaluating cysteamine in
Huntington’s disease.

Verny C(1), Bachoud-Lévi AC(2), Durr A(3), Goizet C(4), Azulay JP(5), Simonin
C(6), Tranchant C(7), Calvas F(8), Krystkowiak P(9), Charles P(3), Youssov K(2),
Scherer C(1), Prundean A(1), Olivier A(1), Reynier P(10), Saudou F(11)(12)(13),
Maison P(14), Allain P(15), von Studnitz E(16), Bonneau D(10); CYST-HD Study
Group.

Author information:
(1)Centre Hospitalier Universitaire d’Angers, Département de Neurologie et UMR
CNRS 6214 – INSERM U1083 et Institut Mitovasc, Angers, France.
(2)Assistance Publique-Hôpitaux de Paris, Centre National de Référence Maladie de
Huntington, Centre Hospitalier Universitaire H. Mondor – A. Chenevier de Créteil
et INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil et Ecole
Normale Supérieure, Institut d’Etudes Cognitives, Paris et Université Paris-Est,
Faculté de Médecine, Créteil, France.
(3)Assistance Publique-Hôpitaux de Paris, Département de Génétique, and Institut
du Cerveau et de la Moelle épinière, Hôpital de la Pitié-Salpêtrière, Paris,
France.
(4)Centre Hospitalier Universitaire de Bordeaux, Hôpital Pellegrin, Service de
Génétique Médicale, Université de Bordeaux, INSERM U1211, Bordeaux, France.
(5)Assistance Publique-Hôpitaux de Marseille, Hôpital de la Timone, Département
de neurologie et de pathologie du mouvement, Institut de neurosciences de la
Timone, UMR 7289 AMU-CNRS, Marseille, France.
(6)Institut de Recherche sur le Cancer de Lille, INSERM UMR837, Centre
Hospitalier Universitaire de Lille, Département de Neurologie et des Mouvements
Anormaux, Lille, France.
(7)Hôpitaux Universitaire de Strasbourg, Hôpital Hautepierre, Service de
Neurologie, Unité des Pathologies du mouvement, Strasbourg, France.
(8)Centre Hospitalier Universitaire Purpan, Centre d’Investigation Clinique,
Toulouse, France.
(9)Centre Hospitalier Universitaire d’Amiens, Département de Neurologie,
Université de Picardie Jules Verne, EA4559, Laboratoire de Neurosciences
Fonctionnelles et Pathologie, Amiens, France.
(10)Centre Hospitalier Universitaire d’Angers, Département de Biochimie et
Génétique et UMR CNRS 6214 – INSERM U1083 et Institut Mitovasc, Angers, France.
(11)Université Grenoble Alpes, Grenoble Institut des Neurosciences, GIN,
Grenoble, France.
(12)INSERM U1216.
(13)Centre Hospitalier Universitaire de Grenoble, Grenoble, France.
(14)INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil, France.
(15)Centre Hospitalier Universitaire d’Angers, Département de Neurologie et UPRES
EA 4638, Laboratoire de Psychologie des Pays de la Loire, Angers, France.
(16)Independent Medical Writing Consultant, Vallejo California, USA.

BACKGROUND: Cysteamine has been demonstrated as potentially effective in numerous
animal models of Huntington’s disease.
METHODS: Ninety-six patients with early-stage Huntington’s disease were
randomized to 1200 mg delayed-release cysteamine bitartrate or placebo daily for
18 months. The primary end point was the change from baseline in the UHDRS Total
Motor Score. A linear mixed-effects model for repeated measures was used to
assess treatment effect, expressed as the least-squares mean difference of
cysteamine minus placebo, with negative values indicating less deterioration
relative to placebo.
RESULTS: At 18 months, the treatment effect was not statistically significant –
least-squares mean difference, -1.5 ± 1.71 (P = 0.385) – although this did
represent less mean deterioration from baseline for the treated group relative to
placebo. Treatment with cysteamine was safe and well tolerated.
CONCLUSIONS: Efficacy of cysteamine was not demonstrated in this study population
of patients with Huntington’s disease. Post hoc analyses indicate the need for
definitive future studies. © 2017 International Parkinson and Movement Disorder
Society.

© 2017 International Parkinson and Movement Disorder Society.

DOI: 10.1002/mds.27010
PMID: 28436572 [Indexed for MEDLINE]

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