Design considerations for efficient prostate cancer chemoprevention trials

Urology. 2001 Apr;57(4 Suppl 1):205-12. doi: 10.1016/s0090-4295(00)00976-6.

Abstract

Prostate cancer, even with its substantial public health impact of 180,400 new cases and 31,900 deaths estimated for 2000, still has a very low annual incidence (0.27% for men 34.4 years and older), which makes designing and conducting efficient prostate cancer prevention trials a challenge. Definitive prevention trials with cancer endpoints, such as the Breast Cancer Prevention Trial (BCPT), Prostate Cancer Prevention Trial (PCPT), and Selenium and Vitamin E Cancer Prevention Trial (SELECT), require long trial duration (up to 12 years) and large sample size (up to 32,400 subjects) to accomplish their objectives. This article discusses design concepts for potential prostate cancer prevention trials that require fewer years, subjects, and resources to complete. Design elements, such as high-risk populations, randomization, surrogate endpoints, including quality-of-life endpoints, masking/blinding, and various clinical/statistical designs (including 1-way layout, all-versus-none, factorial, and adaptive designs), are discussed, along with the ultimate goal of gaining US Food and Drug Administration approval for prostate-cancer preventive agents that can improve public health by reducing prostate cancer incidence and mortality.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Anticarcinogenic Agents / therapeutic use*
  • Biomarkers, Tumor / analysis*
  • Clinical Trials as Topic / methods*
  • Combined Modality Therapy
  • Disease Progression
  • Drug Approval
  • Humans
  • Incidence
  • Male
  • Neoplasm Recurrence, Local
  • Patient Selection
  • Prostate-Specific Antigen / analysis
  • Prostatic Intraepithelial Neoplasia / pathology
  • Prostatic Neoplasms / epidemiology
  • Prostatic Neoplasms / etiology
  • Prostatic Neoplasms / prevention & control*
  • Quality of Life
  • Randomized Controlled Trials as Topic / methods
  • Reproducibility of Results
  • Research Design
  • Risk Factors
  • Sample Size
  • Treatment Outcome
  • United States / epidemiology
  • United States Food and Drug Administration

Substances

  • Anticarcinogenic Agents
  • Biomarkers, Tumor
  • Prostate-Specific Antigen