Phase 0 clinical trials in cancer drug development: From FDA guidance to clinical practice

Robert Kinders, Ralph E. Parchment, Jay Ji, Shivaani Kummar, Anthony J. Murgo, Martin Gutierrez, Jerry Collins, Larry Rubinstein, Oxana Pickeral, Seth M. Steinberg, Sherry Yang, Melinda Hollingshead, Alice Chen, Lee Helman, Robert Wiltrout, Mel Simpson, Joseph E. Tomaszewski, James H. Doroshow

Research output: Contribution to journalReview articlepeer-review

26 Scopus citations


The Food and Drug Administration (FDA) recently introduced the Exploratory Investigational New Drug Guidance to expedite the clinical evaluation of new therapeutic and imaging agents. Early clinical studies performed under the auspices of this guidance, so-called "Phase 0" trials, have been initiated at the National Cancer Institute to integrate qualified pharmacodynamic biomarker assays into first-in-human cancer clinical trials of molecularly targeted agents. The goal of this integration is to perform molecular proof-of-concept investigations at the earliest stage of cancer drug development. Phase 0 trials do not offer any possibility of patient benefit; instead, intensive, real-time pharmacodynamic and pharmacokinetic analyses of patient tumor samples and/or surrogate tissues are performed to inform subsequent trials. Phase 0 studies do not replace formal Phase I drug safety testing and require a substantial investment of resources in assay development early on; however, they offer the promise of more rational selection of agents for further, large-scale development as well as the molecular identification of potential therapeutic failures early in the development process.

Original languageEnglish (US)
Pages (from-to)325-334
Number of pages10
JournalMolecular interventions
Issue number6
StatePublished - Jan 1 2008
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Medicine


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