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6 results

  • 23 - Multiple Sclerosis
  • from Section 6
  • Edited by Bernard Ravina, Jeffrey Cummings, Michael McDermott, R. Michael Poole
  • Book: Clinical Trials in Neurology
  • Published online: 05 May 2012
  • Print publication: 12 April 2012, pp 257-272
  • View abstract

    Summary

    Special trial designs have been developed to distinguish the symptomatic and disease modifying effects of treatment using clinical outcome measures. These designs, termed 'two-period' designs, include the so-called withdrawal and delayed-start (or 'staggered-start') designs and their variations. This chapter describes these study designs in terms of their rationale, assumptions, design features, implementation, statistical analysis, and sample size considerations. It also discusses the important limitations of the designs. Simulation studies using disease progression modeling suggest that the withdrawal design may provide more power than the delayed start design to detect disease-modifying effects of a treatment. A statistical model for data from a complete two-period design assumes that a normally-distributed outcome termed µ 2. There are alternative approaches to evaluating the disease-modifying effects of an intervention that require only a single treatment period.
  • 6 - Measures of neurological impairment and disability in multiple sclerosis
  • from Section II - Clinical trial methodology
  • Edited by Jeffrey A. Cohen, Richard A. Rudick
  • Book: Multiple Sclerosis Therapeutics
  • Published online: 05 December 2011
  • Print publication: 20 October 2011, pp 56-64
  • View extract

    Summary

    This chapter deals with the measurement of impairment or disability for use in multiple sclerosis (MS) clinical trials from a medical model perspective, focusing on global clinical trial outcome measures. It addresses the methodological issues in measuring impairment and disability. The measures of neurological impairment and disability in MS can be grouped into four classes: biological assays, performance measures, rating scales and self-report measures. The Incapacity Status Scale and the multiple sclerosis (MS) Quality of Life Inventory are examples of such self-report measures, also called patient reported outcomes or PROs. Surrogate outcome measures have been studied in a variety of diseases and have been hoped for within the MS community. The development of the Multiple Sclerosis Functional Composite (MSFC) resulted from the analysis of a pooled data set of placebo control groups and natural history study databases.