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Barnett, Helen ; Boix, Oliver ; Kontos, Dimitris ; Jaki, Thomas

Joint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes

Barnett, Helen, Boix, Oliver, Kontos, Dimitris and Jaki, Thomas (2024) Joint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes. Statistics in Biopharmaceutical Research 17 (1), pp. 149-160.

Date of publication of this fulltext: 22 Sep 2025 06:19
Article
DOI to cite this document: 10.5283/epub.77766


Abstract

In Phase I/II dose-finding trials, the objective is to find the Optimal Biological Dose (OBD), a dose that is both safe and shows sufficient activity that maximizes some optimality criterion based on safety and activity. In cancer, treatment is typically given over several cycles, complicating the identification of the OBD as both toxicity and activity outcomes may occur at any point throughout ...

In Phase I/II dose-finding trials, the objective is to find the Optimal Biological Dose (OBD), a dose that is both safe and shows sufficient activity that maximizes some optimality criterion based on safety and activity. In cancer, treatment is typically given over several cycles, complicating the identification of the OBD as both toxicity and activity outcomes may occur at any point throughout the follow up of multiple cycles. In this work we present and assess the Joint TITE-CRM, a model-based design for late onset toxicities and activity based on the well-known TITE-CRM. It is found to be superior to the currently available alternative designs that account for late onset bivariate outcomes, as well as being both intuitive and computationally feasible.



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Details

Item typeArticle
Journal or Publication TitleStatistics in Biopharmaceutical Research
Publisher:Taylor & Francis Online
Volume:17
Number of Issue or Book Chapter:1
Page Range:pp. 149-160
Date20 March 2024
InstitutionsInformatics and Data Science > Department Machine Learning & Data Science > Lehrstuhl für Computational Statistics (Prof. Dr. Thomas Jaki)
Identification Number
ValueType
10.1080/19466315.2024.2333388DOI
KeywordsDose-finding, Late-onset activity, Late-onset toxicities, Model-Based, Phase I trials
Dewey Decimal Classification000 Computer science, information & general works > 004 Computer science
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgPartially
URN of the UB Regensburgurn:nbn:de:bvb:355-epub-777660
Item ID77766

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