Design and Analysis of Phase II Clinical Trials

A predictive probability design for phase II cancer clinical trials.

BACKGROUND Two- or three-stage designs are commonly used in phase II cancer clinical trials. These designs possess good frequentist properties and allow early termination of the trial when the interim data indicate that the experimental regimen is inefficacious. The rigid study design, however, can be difficult to follow exactly because the response has to be evaluated at prespecified fixed num...

Design issues of randomized phase II trials and a proposal for phase II screening trials.

Future progress in improving cancer therapy can be expedited by better prioritization of new treatments for phase III evaluation. Historically, phase II trials have been key components in the prioritization process. There has been a long-standing interest in using phase II trials with randomization against a standard-treatment control arm or an additional experimental arm to provide greater ass...

Design of Phase III Clinical Trials

Modeling Heterogeneity in Phase II Clinical Trials

Problem statement: The common assumption in non-randomized Phase II clinical trials is a homogeneous population with homogeneous response. This assumption is at odds with many trials today; a heterogeneous response due to the existence of subgroups. Approach: In order to examine the effects of heterogeneity on the trial outcome, a systematic platform is developed to quantify the range and class...

ST4241: Design and Analysis of Clinical Trials