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Early clinical trials of chemopreventive and biologic agents: Designs, populations, and endpoints

P Keegan1, B E Loughman

  • 1Division of Clinical Trials Design and Analysis, Office of Therapeutics, Center for Biologics Evaluation and Research, US Food and Drug Administration, Rockville, Maryland 20852-1445, USA. keegan@cber.fda.gov

Urology
|April 11, 2001
PubMed

Insights

Designing cancer chemoprevention trials requires careful consideration of drug mechanisms and available assays. A flexible approach is needed due to diverse agents and targets, as illustrated by R-flurbiprofen for prostate cancer.

Area of Science:

  • Oncology
  • Clinical Pharmacology
  • Drug Development

Background:

  • Optimal design for early-phase cancer chemoprevention trials remains undefined.
  • Heterogeneity of chemopreventive agents and their targeted biological effects necessitates tailored trial designs.
  • Key design factors include proposed mechanisms of action, assay availability for pharmacodynamic effects, and prior clinical experience.

Purpose of the Study:

  • To discuss critical factors for designing initial clinical trials of cancer chemopreventive agents.
  • To present a case study of early clinical assessment for a specific chemopreventive agent.

Main Methods:

  • Review of factors influencing the design of early clinical activity studies for chemopreventive agents.
  • Detailed examination of the early clinical assessment of R-flurbiprofen (E-7869), a non-cytotoxic agent, for prostate cancer chemoprevention.

Main Results:

  • Identified key considerations for designing chemoprevention trials, including drug mechanism, pharmacodynamic assays, and clinical experience.
  • Demonstrated the application of these considerations through the assessment of R-flurbiprofen for prostate cancer.

Conclusions:

  • A one-size-fits-all trial design is unsuitable for cancer chemoprevention due to drug and target variability.
  • A comprehensive approach considering drug properties and assay capabilities is essential for effective early-phase chemoprevention trials.

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