Phase 0 clinical trials: Conceptions and misconceptions

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

Research output: Contribution to journalReview article

Abstract

Phase 0 clinical trials, developed in response to the United States Food and Drug Administration (FDA)s recent exploratory Investigational New Drug (IND) guidance, are intended to expedite the clinical evaluation of new molecular entities. The exploratory IND supports the performance of first-in-human testing of new investigational agents at subtherapeutic doses based on reduced manufacturing and toxicologic requirements, allowing the demonstration of drug-target effects and assessment of pharmacokineticĝ€" pharmacodynamic relationships in humans earlier in clinical development. The objectives of a phase 0 cancer clinical trial are to establish at the very earliest opportunityĝ€"before large numbers of patients have been accrued and exposed to potential drug-associated toxicityĝ€ "whether an agent is modulating its target in a tumor, and consequently whether further clinical development is warranted. We review here the fundamental requirements of clinical studies conducted under an exploratory IND and address some common misconceptions regarding oncologic phase 0 trials.

Original languageEnglish (US)
Pages (from-to)133-137
Number of pages5
JournalCancer Journal
Volume14
Issue number3
DOIs
StatePublished - May 1 2008

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Keywords

  • Clinical trial
  • Exploratory IND
  • Oncology
  • Pharmacodynamics
  • Phase 0

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Kummar, S., Rubinstein, L., Kinders, R., Parchment, R. E., Gutierrez, M. E., Murgo, A. J., Ji, J., Mroczkowski, B., Pickeral, O. K., Simpson, M., Hollingshead, M., Yang, S. X., Helman, L., Wiltrout, R., Collins, J., Tomaszewski, J. E., & Doroshow, J. H. (2008). Phase 0 clinical trials: Conceptions and misconceptions. Cancer Journal, 14(3), 133-137. https://doi.org/10.1097/PPO.0b013e318172d6f3