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Clinical Trial Design in the Age of Molecular Profiling

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Molecular Profiling

Part of the book series: Methods in Molecular Biology ((MIMB,volume 823))

Abstract

The accelerating science of molecular profiling has necessitated a rapid evolution in clinical trial design. Traditional clinical research begins with Phase I studies to characterize dose-limiting toxicities and defines maximally tolerated doses of drugs in small numbers of patients. Traditional Phase II studies test these drugs at the doses discovered during Phase I drug development in small numbers of patients evaluating efficacy and safety. Phase III studies test new therapies to demonstrate improved activity or improved tolerability compared with a standard of care regimen or a placebo. The rapid advances in the understanding of signal transduction, and the identification of new potential diagnostic and therapeutic targets, now require the design and implementation of molecular clinical trials that are very different than traditional Phase I, II, or III trials. The main differentiating factor is the use of a molecular end point to stratify a subset of patients to receive a specific treatment regimen. This chapter focuses on the issues surrounding (a) the definition of clinical end points and the assessment of tumor response; (b) clinical trial design models to define the targeted pathway; and (c) the need for appropriate biomarkers to monitor the response.

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Author information

Authors and Affiliations

  1. Virginia Cancer Specialists, Fairfax, VA, USA

    Alexander Spira

  2. Inova Fairfax Hospital Cancer Center, Fairfax, VA, USA

    Kirsten H. Edmiston

Authors
  1. Alexander Spira
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  2. Kirsten H. Edmiston
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Corresponding author

Correspondence to Kirsten H. Edmiston .

Editor information

Editors and Affiliations

  1. Center for Applied Proteomics and, George Mason University, University Blvd. 10900, Manassas, 20110, Virginia, USA

    Virginia Espina

  2. Center for Applied Proteomics &, George Mason University, University Blvd. 10900, Manassas, 20110, Virginia, USA

    Lance A. Liotta

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Spira, A., Edmiston, K.H. (2012). Clinical Trial Design in the Age of Molecular Profiling. In: Espina, V., Liotta, L. (eds) Molecular Profiling. Methods in Molecular Biology, vol 823. Humana Press. https://doi.org/10.1007/978-1-60327-216-2_2

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  • DOI: https://doi.org/10.1007/978-1-60327-216-2_2

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  • Publisher Name: Humana Press

  • Print ISBN: 978-1-60327-215-5

  • Online ISBN: 978-1-60327-216-2

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