Two-stage Adaptive Enrichment Designs with Survival Outcomes and Adjustment for Misclassification in Predictive Biomarkers.

ABSTRACT: Biomarker enrichment clinical trial designs are versatile tools to assess the treatment effect and increase the efficiency of clinical trials. In this paper, we propose a two-stage enrichment clinical trial design with survival outcomes, and consider the situation where the biomarker assay and classification are possibly subject to errors. Specifically, the first stage is a randomized design, stratified by the biomarker appeared status. Depending on the result of the interim analysis and a pre-specified futility criterion, the second stage can be either enriched with only the biomarker appeared positive patients, or remain as the stratified design with both biomarker appeared positive and biomarker appeared negative patients. Compared to continuous and binary outcomes, test statistics to a

SUBMITTER: Chen Y 

PROVIDER: S-EPMC12530146 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature