Phase II clinical trials in oncology: are we hitting the target?

Expert Rev Anticancer Ther. 2010 Mar;10(3):427-38. doi: 10.1586/era.09.178.

Abstract

The number of novel and molecularly targeted agents in the last decade that need screening for preliminary efficacy in Phase II trials has increased. Many of these agents have a cytostatic mode of action that is difficult to assess using traditional Phase II designs. These new agents require detailed evaluation to optimize their dosing, to evaluate their effects on their target and to define early markers that predict for a definitive benefit. This review focuses on the options for Phase II trial designs. The different end points, single versus multiarm and randomized designs, the use of biomarkers and Bayesian approaches are also reviewed. The final design chosen will depend on the characteristics and circumstances of each individual study.

Publication types

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

MeSH terms

  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / pharmacology*
  • Bayes Theorem
  • Biomarkers, Tumor / analysis
  • Clinical Trials, Phase II as Topic / methods*
  • Drug Delivery Systems
  • Endpoint Determination
  • Humans
  • Neoplasms / drug therapy*
  • Research Design

Substances

  • Antineoplastic Agents
  • Biomarkers, Tumor