STOPPING RULES FOR LONG TERM CLINICAL TRIALS BASED ON TWO CONSECUTIVE: REJECTIONS OF THE NULL HYPOTHESIS

基于两次连续拒绝零假设的长期临床试验终止规则

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Abstract

A strategy for stopping long term randomized clinical trials with time-to-event as a primary outcome measure has been considered using the criteria requiring multiple consecutive (or non-consecutive) rejections at a specified α-level that controls against elevation of type I error. The procedure using two consecutive rejections is presented in this work along with the corresponding α-levels for the interim tests. The boundary cutoff values for these interim levels were determined based on an overall pre-specified test size and were calculated using multi-dimensional integration and/or simulations. The reduction in the interim α-level values that is required to maintain the experiment-wise error rate is found to be modest. The power of the test is evaluated under various alternative accrual and hazard patterns. This procedure provides a more realistic stopping rule in large multi-center trials where it may be undesirable to terminate a trial unless a sustained effect has been demonstrated.

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