The power of phase II end-points for different possible mechanisms of action of an experimental treatment.

ABSTRACT:

Background

The high failure rate in phase III oncology trials is partly because the signal obtained from phase II trials is often weak. Several papers have considered the appropriateness of various phase II end-points for individual trials, but there has not been a systematic comparison using simulated data to determine which end-point should be used in which situation.

Methods

In this paper we carry out simulation studies to compare the power of several Response Evaluation Criteria in Solid Tumours (RECIST) response-based end-points for one-arm and two-arm trials, together with progression-free survival (PFS) and testing the tumour-shrinkage directly for two-arm trials. We consider six scenarios: (1) short-term cytotoxic therapy; (2) continuous cytotoxic therapy; (3+4) cyto

SUBMITTER: Wason JM 

PROVIDER: S-EPMC4435668 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature