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Multiple Sclerosis: Study Design, Sample Sizes and Pitfalls

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Clinical Trials in Neurology

Abstract

Rapid growth in the number, scale and quality of clinical trials in multiple sclerosis (MS) has occurred over the last decade. The recent surge of interest has resulted from a number of developments. Firstly, basic scientific inquiry in such areas as cytokine research, T cell receptor gene recombination and adhesion molecule interactions has suggested novel methods of treatment, including the use of immunoregulatory cytokines (e.g. interferon beta, transforming growth factor beta), T cell or T cell receptor peptide vaccination, and monoclonal antibodies to adhesion cell antigens expressed on the surface of lymphocytes and endothelial cells so as to block their interaction [1]. Secondly, a certain degree of optimism has developed following the report of positive results in three phase III trials of therapeutic agents [2–4], which have now been demonstrated to reduce attacks. One agent, interferon beta-la (Avonex), reduced short-term progression of neurological impairment as determined by change in terms of the Expanded Disability Status Scale (EDSS). Finally, rapid advances in magnetic resonance (MR) technology have provided a sensitive, objective, quantitative, and easily blind method for detecting treatment effects in phase I and II trials in a fraction of the time necessary for a definitive phase III clinical trial. MR technology has been proposed and widely accepted to be the primary outcome of choice for screening potential therapeutic agents for efficacy in phase I and II clinical trials [5].

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Authors
  1. B. G. Weinshenker
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  2. J. H. Noseworthy
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Editors and Affiliations

  1. Neuromuscular Unit, West London Neurosciences Centre, 1st Floor Pilot Wing, Charing Cross Hospital, Fulham Palace Road, W6 8RF, London, UK

    Roberto J. Guiloff MD, FRCP (Consultant Neurologist) (Consultant Neurologist)

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© 2001 Springer-Verlag London

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Weinshenker, B.G., Noseworthy, J.H. (2001). Multiple Sclerosis: Study Design, Sample Sizes and Pitfalls. In: Guiloff, R.J. (eds) Clinical Trials in Neurology. Springer, London. https://doi.org/10.1007/978-1-4471-3787-0_31

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  • DOI: https://doi.org/10.1007/978-1-4471-3787-0_31

  • Publisher Name: Springer, London

  • Print ISBN: 978-1-84996-856-0

  • Online ISBN: 978-1-4471-3787-0

  • eBook Packages: Springer Book Archive

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