Randomized Phase II Cancer Clinical Trials with a Prospective Control on Binary Endpoints (I): Two-Sample Binomial Test

As an effort to speed the assessment of new therapies, a phase II clinical trial traditionally recruits a small number of patients only to the experimental therapy arm to be compared to a historical control. This implies that the traditional single-arm phase II trials are appropriate only when reliable and valid data for an existing standard therapy are available for the same patient population. Furthermore, the response assessment method used for the historical control should be identical to the one that will be used for a new study.