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Abstract:
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We introduce phase II single-arm two-stage designs for safety by generalizing phase II single-arm two-stage designs in terms of both safety and efficacy endpoints. In phase II single-arm trials, one of the most popular methods is Simon's two-stage and alike designs with an efficacy endpoint where the null response rate is less than its corresponding alternative rate. Phase II single-arm trials often have the rate of adverse events or toxicity of treatments as the primary endpoint for safety (or tolerability) where the null response rate is greater than the alternative rate. All existing designs for phase II single-arm trials are dedicated mostly to the trials with an efficacy endpoint, however, and no practical two-stage design is available for a safety endpoint yet. Besides, no detailed description on how to use the existing efficacy two-stage designs has been provided for the safety endpoint, discouraging investigators from the use of two-stage designs for safety in practice. Therefore, we introduce generalized two-stage designs for phase II single-arm trials that can be used for both safety and efficacy endpoints, followed by theoretical justifications and real applications.
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