Seamlessly expanding a randomized phase II trial to phase III
Seamlessly expanding a randomized phase II trial to phase III
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DOI：
10.1111/j.0006-341x.2002.00823.x
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发表时间：
2002-12-01
期刊：
影响因子：
1.9
通讯作者：
Berry, DA
中科院分区：
文献类型：
--
作者：
Inoue, LYT;Thall, PF;Berry, DA
序贯贝叶斯II/III期设计被提出用于临床对比试验。该设计基于生存时间和可能与生存相关的离散早期事件，并假设为参数混合模型。第11阶段涉及少量的中心。在整个治疗过程中，患者在不同的治疗之间随机进行，顺序决定是基于得出实验治疗优越性的预测概率。是否提前停止、继续或转移到第三阶段在第二阶段反复评估第三阶段当更多的机构被纳入正在进行的第二阶段试验时第三阶段开始。在非小细胞肺癌试验背景下的模拟研究表明，与传统的分组序贯III期设计相比，所提出的方法保持了总体规模和功率，同时通常需要显著更小的样本量和更短的试验持续时间。
A sequential Bayesian phase II/III design is proposed for comparative clinical trials. The design is based on both survival time and discrete early events that may be related to survival and assumes a parametric mixture model. Phase 11 involves a small number of centers. Patients are randomized between treatments throughout, and sequential decisions are based on predictive probabilities of concluding superiority of the experimental treatment. Whether to stop early, continue, or shift into phase III is assessed repeatedly in phase II Phase III begins when additional institutions are incorporated into the ongoing phase II trial. Simulation studies in the context of a non-small-cell lung cancer trial indicate that the proposed method maintains overall size and power while usually requiring substantially smaller sample size and shorter trial duration when compared with conventional group-sequential phase III designs.