Unified exact design with early stopping rules for single arm clinical trials with multiple endpoints. (July 2021)

• Record Type: Journal Article
• Authors: Wei, Wei; Esserman, Denise; Kane, Michael; Zelterman, Daniel
• Is Part Of: Statistical methods in medical research. Volume 30:Number 7(2021)
• Page Start: 1575
• Page End: 1588
• Publication Date: 2021-07
• Subjects: Go/no go decisions -- multiple endpoints -- biomarker endpoints -- conditional power -- early phase; Medicine -- Research -- Statistical methods -- Periodicals; Research -- Periodicals; Review Literature -- Periodicals; Statistics -- methods -- Periodicals; Médecine -- Recherche -- Méthodes statistiques -- Périodiques; 610.727
• DOI: 10.1177/09622802211013062
• Languages: English
• ISSNs: 0962-2802

Abstract

Adaptive designs are gaining popularity in early phase clinical trials because they enable investigators to change the course of a study in response to accumulating data. We propose a novel design to simultaneously monitor several endpoints. These include efficacy, futility, toxicity and other outcomes in early phase, single-arm studies. We construct a recursive relationship to compute the exact probabilities of stopping for any combination of endpoints without the need for simulation, given pre-specified decision rules. The proposed design is flexible in the number and timing of interim analyses. A R Shiny app with user-friendly web interface has been created to facilitate the implementation of the proposed design.