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

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.

Keywords::

• biomarker
• cytostatic agent
• end point
• enrichment
• Phase II study
• randomization
• targeted therapy

Financial & competing interests disclosure

M-K Ang is supported by the Singapore Millenium Foundation Scholarship. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.