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U.S. National Institutes of Health
Cancer Diagnosis Program Cancer Imaging Program Cancer Therapy Evaluation Program Developmental Therapeutics Program Radiation Research Program Translational Research Program Biometric Research Branch Office of Cancer Complementary and Alternative Medicine
Last Updated: 04/25/2012

Testing a New Oncology Drug Development Paradigm - Section 4

The distinctive features of phase 0 studies are:

  • First-in-human, with single or combination drugs
  • Molecules from CCR, academia, small biotech
  • Small patient numbers (6–10); joint CCR-DCTD clinical trial effort performed in the Clinical Research Center
  • Provide PK/PD data to support rapid future dose escalation based on extensive preclinical toxicology and target effect studies
  • Initial target assay development and drug/target assessments (primary and surrogate, imaging and molecular expression)
  • Preliminary toxicity evaluation in humans
  • Inform subsequent broad phase I/II trials
  • High throughput of trials, each completed in three to six months
  • Preliminary toxicity evaluation in humans

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