Abstract:
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Conventional phase II clinical trials use either a single-arm or two-arm comparison scheme to examine the experimental drug's therapeutic effects. Both single-arm and multi-arm evaluations have their own merits; for example, single-arm phase II trials are easy to conduct and often require a smaller sample size, while multi-arm trials are randomized and typically lead to a more objective comparison. To bridge the single- and two-arm schemes in phase II clinical trials, we propose a two-stage design, in which the first stage takes a single-arm comparison of the experimental drug with the standard response rate (no concurrent treatment) and the second stage imposes a multi-arm comparison of the experimental drug with an active control arm. The design is calibrated using a new concept, the detectable treatment difference, to balance the tradeoffs between adaptation, power, and sample size. We conduct extensive simulation studies to examine the operational characteristics of our proposed method, and give an illustrative example of our design.
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