Many oncology phase II trials are single arm studies designed to screen novel treatments based on efficacy outcome. Efficacy is often assessed as an ordinal variable based on a level of response of solid tumors with four categories: complete response, partial response, stable disease and progression. We describe a two-stage design for a single-arm phase II trial where the primary objective is to test the rate of tumor response defined as complete plus partial response, and the secondary objective is to estimate the rate of disease control defined as tumor response plus stable disease. Since the goal is to estimate the disease control rate, the trial is not stopped for futility after the first stage if the disease control rate is promising. The new design can be generated using easy-to-use software that is available at http://cancer.unc.edu/biostatistics/program/ivanova/.

中文翻译：

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具有宽松无效停止的 II 期肿瘤学试验的两阶段设计

许多肿瘤学 II 期试验是单臂研究，旨在根据疗效结果筛选新疗法。疗效通常被评估为基于实体瘤反应水平的有序变量，分为四类：完全反应、部分反应、稳定疾病和进展。我们描述了单臂 II 期试验的两阶段设计，其中主要目标是测试定义为完全加部分反应的肿瘤反应率，次要目标是估计定义为肿瘤反应的疾病控制率加上病情稳定。由于目标是估计疾病控制率，如果疾病控制率有希望，试验不会在第一阶段后因无效而停止。可以使用 http://cancer.unc 上提供的易于使用的软件生成新设计。