Multi-stage design in phase II clinical trials for anti-cancer drug development / 肿瘤

ABSTRACT

In phase II clinical trials, multiple kinds of tumors were treated with anti-cancer drugs at different dosages and through different administration pathways to exclude the ineffective dosages and screen sensitive tumor (s) for further study. When expectant effects are not in fact observed in the experimental groups, it is essential to terminate the trials to minimize exposure of subjects to ineffective interventions. The trials are usually carried out with a multi-stage design. The present paper introduces the multi-stage design including the statistic principles and design ideas of single-stage, two-stage and three-stage design, provided the sample sizes needed in clinical trials at different stages, and estimated the value of early termination. All are illustrated with an example.