On Optimal Subset Designs for Phase II Clinical Trials With Both Total Response and Disease Control

Document Type

Article

Publication Date

12-1-2021

Identifier/URL

40915884 (Pure)

Abstract

Phase II clinical trials in oncology are used to initially evaluate the therapeutic efficacy of a new treatment. In the past, the total response was a frequently used endpoint to access the effectiveness of the treatment. When the total response is modest, clinicians may also be interested in disease control (defined as the total response or stable disease) since it may better predict clinical outcomes. Thus, formally testing both the total response and disease control in a phase II trial has an important clinical implication. In this paper, we propose a new method to construct optimal subset designs for one-stage and two-stage phase II clinical trials with two binary endpoints, i.e. the total response and disease control. A new set of hypotheses under the framework of intersection-union tests is provided in which the treatment is considered promising if both the total response and disease control are good. We show that the power function for each test in a large family of tests is nondecreasing in both the total response rate and disease control rate; identify the parameter configurations at which the maximum Type I error rate and minimum power are achieved and derive level-x tests. We also provide optimal one-stage designs with the minimum sample size and optimal two-stage designs with the least expected total sample size.

Comments

Publisher Copyright: © 2021 Elsevier B.V.

DOI

10.1016/j.jspi.2021.02.007

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