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M2 A randomised, double-blind, placebo-controlled trial evaluating cysteamine in huntington’s disease
  1. Dominique Bonneau1,
  2. Anne-Catherine Bachoud-Lévi2,
  3. Alexandra Durr3,
  4. Cyril Goizet4,
  5. Jean-Philippe Azulay5,
  6. Clémence Simonin6,
  7. Christine Tranchant7,
  8. Fabienne Calvas7,8,
  9. Pierre Krystkowiak9,
  10. Audrey Olivier10,
  11. Frédéric Saudou11,
  12. Patrick Maison12,
  13. Philippe Allain13,
  14. Christophe Verny10
  1. 1Centre Hospitalier Universitaire d’Angers, Département de Biochimie et Génétique et UMR CNRS 6214 – INSERM U1083 et Institut Mitovasc, Centre Hospitalier Universitaire d’Angers, Angers, France
  2. 2Assistance Publique-Hôpitaux de Paris, Centre National de Référence Maladie de Huntington, Centre Hospitalier Universitaire H. Mondor 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. 3Université Pierre et Marie Curie et Assistance Publique-Hôpitaux de Paris, Institut du Cerveau et de la Moelle épinière, Hôpital de la Pitié-Salpêtrière, Paris, France
  4. 4Centre Hospitalier Universitaire de Bordeaux, Hôpital Pellegrin, Service de Génétique Médicale, Université de Bordeaux, INSERM U1211, Bordeaux, France
  5. 5Assistance 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. 6Institut de Recherche sur le Cancer de Lille, INSERM UMR837, Centre Hospitalier Universitaire de Lille, Département de Neurologie et des Mouvements Anormaux, Lille, France
  7. 7Hôpitaux Universitaire de Strasbourg, Hôpital Hautepierre, Service de Neurologie, Unité des Pathologies du mouvement, Strasbourg, France
  8. 8Centre Hospitalier Universitaire Purpan, Centre d’Investigation Clinique, Toulouse, France
  9. 9Centre Hospitalier Universitaire d’Amiens, Département de Neurologie, Université de Picardie Jules Verne, EA4559, Laboratoire de Neurosciences Fonctionnelles et Pathologie, Amiens, France
  10. 10Centre Hospitalier Universitaire d’Angers, Département de Neurologie et UMR CNRS 6214 – INSERM U1083 et Institut Mitovasc, Centre Hospitalier Universitaire d’Angers, Angers, France
  11. 11Université Grenoble Alpes, Institut des Neurosciences GIN INSERM U836, Centre Hospitalier Universitaire de Grenoble, Grenoble, France
  12. 12INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Créteil, France
  13. 13Centre Hospitalier Universitaire d’Angers, Département de Neurologie et UPRES EA 4638, Laboratoire de Psychologie des Pays de la Loire, Angers, France


Background Cysteamine has been demonstrated as potentially neuroprotective in numerous animal models of Huntington’s disease (HD).

Methods Ninety-six early-stage HD patients were randomised to a double-blind trial to receive 600 mg delayed-release cysteamine bitartrate (DR-CB) (51) or placebo (45) twice a day for 18 months.

The primary endpoint was the change from baseline in the Unified Huntington’s Disease Rating Scale (UHDRS) Total Motor Score (TMS) at 18 months. Key secondary endpoints included other UHDRS scales: total functional capacity, independence, cognition and behaviour. Results are expressed as the least-squares mean (LSM) treatment-effect difference of DR-CB minus placebo; negative values indicating less deterioration relative to placebo.

Results The mean baseline TMS (± SD) was 24.2 (10.7) (5 to 53 points). The 18 month treatment-effect (LSM ± standard error [± SE]) was not statistically significant: −1.5 (1.71) points (95% CI: −4.9; 1.9; p = 0.385); representing less mean (± SE) increase (deterioration) from baseline in DR-CB- compared with placebo-treated patients: 4.9 (1.17) versus 6.4 (1.25) points, respectively. No significant treatment-effects were observed for key secondary endpoints. DR-CB was safe and well-tolerated.

Post-hoc analyses dichotomized by median baseline TMS (<23/≥23 points) indicated a statistically significant DR-CB treatment-effect (± SE): −4.7 (2.18) points (p = 0.038) in patients with baseline TMS ≥ 23 points.

Interpretation Statistical significance was not observed for the primary endpoint. However, post-hoc analysis indicated statistical significance in patients with more severe baseline motor impairment. Further studies involving a larger number of patients are warranted.

Trial registration EudraCT Number: 2010–019444-39

  • Cysteamine
  • Clinical trial

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