Unified exact design with early stopping rules for single arm clinical trials with multiple endpoints
- 23 June 2021
- journal article
- research article
- Published by SAGE Publications in Statistical Methods in Medical Research
- Vol. 30 (7), 1575-1588
- https://doi.org/10.1177/09622802211013062
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.Funding Information
- Center for Information Technology (CTSA Grant Number UL1TR001863, P30-CA16359, P50-CA121974, P50-CA196530, R01-CA168733, R01-CA177719, R01-CA223481, R01-ES005775, R35CA197574, R41-A120546, U01-CA235747, U48-DP005023)
- Pilot Grant from Yale Cancer Center
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