Two-stage Optimal Designs with Survival Endpoint when the Follow-up Time is Restricted

Overview

Journal BMC Med Res Methodol

Publisher Biomed Central

Specialties General Medicine
Health Services

Date 2019 Apr 5

PMID 30943896

Citations 8

Authors

Guogen Shan

Hua Zhang

Affiliations

Soon will be listed here.

Abstract

Background: Survival endpoint is frequently used in early phase clinical trials as the primary endpoint to assess the activity of a new treatment. Existing two-stage optimal designs with survival endpoint either over estimate the sample size or compute power outside the alternative hypothesis space.

Methods: We propose a new single-arm two-stage optimal design with survival endpoint by using the one-sample log rank test based on exact variance estimates. This proposed design with survival endpoint is analogous to Simon's two-stage design with binary endpoint, having restricted follow-up.

Results: We compare the proposed design with the existing two-stage designs, including the two-stage design with survival endpoint based on the nonparametric Nelson-Aalen estimate, and Simon's two-stage designs with or without interim accrual. The new design always performs better than these competitors with regards to the expected total study length, and requires a smaller expected sample size than Simon's design with interim accrual.

Conclusions: The proposed two-stage minimax and optimal designs with survival endpoint are recommended for use in practice to shorten the study length of clinical trials.

Citing Articles

Continuity corrected score confidence interval for the difference in proportions in paired data.

Chang P, Liu R, Hou T, Yan X, Shan G J Appl Stat. 2024; 51(1):139-152.

PMID: 38179158 PMC: 10763857. DOI: 10.1080/02664763.2022.2118245.

Response adaptive randomization design for a two-stage study with binary response.

Shan G J Biopharm Stat. 2023; 33(5):575-585.

PMID: 36735855 PMC: 10397367. DOI: 10.1080/10543406.2023.2170399.

Monte Carlo cross-validation for a study with binary outcome and limited sample size.

Shan G BMC Med Inform Decis Mak. 2022; 22(1):270.

PMID: 36253749 PMC: 9578204. DOI: 10.1186/s12911-022-02016-z.

Effects of dose change on the success of clinical trials.

Shan G, Ritter A, Miller J, Bernick C Contemp Clin Trials Commun. 2022; 30:100988.

PMID: 36117568 PMC: 9478360. DOI: 10.1016/j.conctc.2022.100988.

Conservative confidence intervals for the intraclass correlation coefficient for clustered binary data.

Shan G J Appl Stat. 2022; 49(10):2535-2549.

PMID: 35757040 PMC: 9225324. DOI: 10.1080/02664763.2021.1910939.

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