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Clinical Trial Design for Myasthenia Gravis

  • Radwa Aly
  • Gary R. Cutter
  • Henry J. Kaminski
Chapter
Part of the Current Clinical Neurology book series (CCNEU)

Abstract

After an evidence base composed largely of retrospective case series, open-label studies, and expert opinions, evaluation of therapeutics for myasthenia gravis (MG) has become much more rigorous using randomized clinical trial methodologies. Advances in immunotherapeutics and the biological basis of MG have accelerated the number of drugs under development for MG leading to a greater necessity to optimize approaches for evaluation of new treatments. The design of clinical trials involves a multidisciplinary approach encompassing the disease and its characteristics, the drug or treatment and its putative mechanism of action, the inclusion and exclusion criteria, the endpoints, the logistics and duration of the study, and the ability to recruit and complete the trial in a reasonable time frame. The chapter provides a broad overview of clinical trial development for MG.

Keywords

Myasthenia gravis Clinical trials Phase II Phase II Phase III Phase IV Adaptive design Bayesian approach Classical approach Quantitative myasthenia gravis score Mg-ADL MG composite 

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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  • Radwa Aly
    • 1
  • Gary R. Cutter
    • 2
  • Henry J. Kaminski
    • 1
  1. 1.Department of NeurologyThe George Washington UniversityWashington, DCUSA
  2. 2.Department of BiostatisticsUniversity of Alabama at BirminghamBirminghamUSA

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