Clinical trial design in the age of molecular profiling

Alexander Spira, Kirsten H. Edmiston

Research output: Chapter in Book/Report/Conference proceedingChapter

3 Scopus citations


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.

Original languageEnglish (US)
Title of host publicationMolecular Profiling
Subtitle of host publicationMethods and Protocols
EditorsVirginia Espina, Lance Liotta
Number of pages16
StatePublished - 2012
Externally publishedYes

Publication series

NameMethods in Molecular Biology
ISSN (Print)1064-3745


  • Biomarkers
  • Clinical trial
  • Design
  • End points
  • Imaging
  • Pharmacodynamics
  • Tumor

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics


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