Randomized phase III clinical trial designs for targeted agents.

Publication Type:

Journal Article


Clinical cancer research : an official journal of the American Association for Cancer Research, Volume 14, Issue 14, p.4358-67 (2008)


2008, Antineoplastic Agents, Clinical Trials, Phase III as Topic, Drug Delivery Systems, Humans, Neoplasms, Public Health Sciences Division, Randomized Controlled Trials as Topic, Research Design, Tumor Markers, Biological


Cancer therapies with mechanisms of action which are very different from the more conventional chemotherapies are now being developed. In this article, we investigate the performance of several phase III clinical trial designs, both for testing the overall efficacy of a targeted agent and for testing its efficacy in a subgroup of patients with a tumor marker present. We study different designs and different underlying scenarios assuming continuous markers, and assess the trade-off between the number of patients on the study and the effectiveness of treatment in the subgroup of marker-positive patients.