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Phase 0 clinical trials: recommendations from the Task Force on Methodology for the Development of Innovative Cancer

Shivaani Kummar1, James H Doroshow, Joseph E Tomaszewski

  • 1Center for Cancer Research, National Cancer Institute, NIH, Bethesda, MD 20892, USA.

European Journal of Cancer (Oxford, England : 1990)
|December 19, 2008
PubMed

Insights

Phase 0 clinical trials for innovative cancer therapies are debated due to lack of patient benefit. However, these trials offer early human pharmacokinetic and pharmacodynamic data crucial for drug development decisions.

Area of Science:

  • Oncology
  • Pharmacology
  • Clinical Trial Design

Background:

  • The Methodology for the Development of Innovative Cancer Therapies (MDICT) task force convened to guide the development of novel anticancer agents, focusing on targeted therapies.
  • The task force evaluated the utility, design, and application of Phase 0 clinical trials within anticancer drug development.

Framework:

  • Phase 0 trials, while non-therapeutic, provide early human pharmacokinetic and pharmacodynamic data.
  • The lack of direct clinical benefit to patients participating in Phase 0 trials makes their role controversial.

Implementation:

  • Generating early human data from Phase 0 trials can significantly aid in designing subsequent clinical development phases.
  • A 'decision chart' is recommended to help investigators and sponsors determine the suitability of agents for Phase 0 evaluation.

Implications:

  • Phase 0 trials can optimize decision-making processes in anticancer drug development.
  • This guidance supports the efficient progression of innovative targeted cancer therapies.

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