Design optimization for clinical trials in early-stage manifest Huntington's disease

Author:

Frost Chris1,Mulick Amy1,Scahill Rachael I.2,Owen Gail2,Aylward Elizabeth3,Leavitt Blair R.4,Durr Alexandra56,Roos Raymund A. C.7,Borowsky Beth89,Stout Julie C.10,Reilmann Ralf111213,Langbehn Douglas R.14,Tabrizi Sarah J.2,Sampaio Cristina8,

Affiliation:

1. Department of Medical Statistics; London School of Hygiene and Tropical Medicine; London UK

2. Huntington's Disease Centre, UCL Institute of Neurology; Department of Neurodegenerative Disease, University College London; London UK

3. Center for Integrative Brain Research; Seattle Children's Research Institute; Seattle Washington USA

4. Department of Medical Genetics; University of British Columbia; Vancouver British Columbia Canada

5. Brain and Spine Institute, INSERM U1127, Centre National de la Recherche Scientifique, UMR7225; Sorbonne Universités, University Pierre and Marie Curie, Paris VI UMR_S1127; Paris France

6. Assistance Publique - Hôpitaux de Paris, Genetic Department; Pitié -Salpêtrière University Hospital; Paris France

7. Department of Neurology; Leiden University Medical Centre; Leiden The Netherlands

8. CHDI Management, CHDI Foundation; Princeton New Jersey USA

9. Clinical Development, Neurodegenerative Diseases; Teva Pharmaceuticals; Malvern Pennsylvania USA

10. School of Psychological Sciences; Monash University; Melbourne Victoria Australia

11. George Huntington Institute; Muenster Germany

12. Institute for Clinical Radiology; University of Muenster; Muenster Germany

13. Department of Neurodegenerative Diseases and Hertie-Institute for Clinical Brain Research; University of Tuebingen; Tuebingen Germany

14. Department of Psychiatry; University of Iowa; Iowa City Iowa USA

Funder

Cure Huntington’s Disease Initiative (CHDI)

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Reference36 articles.

1. Therapeutic interventions for disease progression in Huntington's disease;Mestre;Cochrane Database Syst Rev,2009

2. Clinical trials in Huntington's disease: interventions in early clinical development and newer methodological approaches;Sampaio;Mov Disord,2014

3. Modelling and simulation in the pharmaceutical industry-some reflections;Burman;Pharm Stat,2011

4. Critical Path Institute U.S. Food and Drug Administration and European Medicines Agency Reach Landmark Decisions on Critical Path Institute's Clinical Trial Simulation Tool for Alzheimer's Disease 2013 http://c-path.org/wp-content/uploads/2014/03/US-FDA-EMA-agency-reach-landmark-decisions-C-Path-clinical-trial-simulation-tool-for-alzheimers-disease.pdf

5. Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data;Tabrizi;Lancet Neurol,2009

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