Cabarrou, Bastien, Leconte, Eve, Sfumato, Patrick, Boher, Jean-Marie and Filleron, Thomas (2022) A stratified adaptive two-stage design with co-primary endpoints for phase II clinical oncology trials. BMC Medical Research Methodology, vol. 22 (n° 278).

Full text not available from this repository.
Identification Number : 10.1186/s12874-022-01748-w


Given the inherent challenges of conducting randomized phase III trials in older cancer patients, single-arm phase II trials which assess the feasibility of a treatment that has already been shown to be effective in a younger population may provide a compelling alternative. Such an approach would need to evaluate treatment feasibility based on a composite endpoint that combines multiple clinical dimensions and to stratify older patients as fit or frail to account for the heterogeneity of the study population to recommend an appropriate treatment approach. In this context, stratified adaptive two-stage designs for binary or composite endpoints, initially developed for biomarker studies, allow to include two subgroups whilst maintaining competitive statistical performances. In practice, heterogeneity may indeed affect more than one dimension and incorporating co-primary endpoints, which independently assess each individual clinical dimension, would therefore appear quite pertinent. The current paper presents a novel phase II design for co-primary endpoints which takes into account the heterogeneity of a population.

Item Type: Article
Language: English
Date: 26 October 2022
Refereed: Yes
Divisions: TSE-R (Toulouse)
Site: UT1
Date Deposited: 04 Apr 2023 07:55
Last Modified: 04 Apr 2023 07:55
OAI Identifier:
View Item