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Clinical Trial Design for Myasthenia Gravis

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Myasthenia Gravis and Related Disorders

Part of the book series: Current Clinical Neurology ((CCNEU))

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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.

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Authors and Affiliations

  1. Department of Neurology, The George Washington University, Washington, DC, USA

    Radwa Aly MS & Henry J. Kaminski MD

  2. Department of Biostatistics, University of Alabama at Birmingham, Birmingham, AL, USA

    Gary R. Cutter PhD

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  1. Radwa Aly MS
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  2. Gary R. Cutter PhD
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  3. Henry J. Kaminski MD
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Correspondence to Henry J. Kaminski MD .

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Editors and Affiliations

  1. Department of Neurology, George Washington University, Washington, DC, USA

    Henry J. Kaminski

  2. Department of Pharmacology and Physiology, George Washington University, Washington, DC, USA

    Linda L. Kusner

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Aly, R., Cutter, G.R., Kaminski, H.J. (2018). Clinical Trial Design for Myasthenia Gravis. In: Kaminski, H., Kusner, L. (eds) Myasthenia Gravis and Related Disorders. Current Clinical Neurology. Humana Press, Cham. https://doi.org/10.1007/978-3-319-73585-6_21

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  • DOI: https://doi.org/10.1007/978-3-319-73585-6_21

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