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Jaki, Thomas ; Barnett, Helen ; Titman, Andrew ; Mozgunov, Pavel

A seamless Phase I/II platform design with a time-to-event efficacy endpoint for potential COVID-19 therapies

Jaki, Thomas , Barnett, Helen, Titman, Andrew und Mozgunov, Pavel (2024) A seamless Phase I/II platform design with a time-to-event efficacy endpoint for potential COVID-19 therapies. Statistical Methods in Medical Research 33 (11-12), S. 2115-2130.

Veröffentlichungsdatum dieses Volltextes: 22 Sep 2025 06:25
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.77767


Zusammenfassung

In the search for effective treatments for COVID-19, the initial emphasis has been on re-purposed treatments. To maximize the chances of finding successful treatments, novel treatments that have been developed for this disease in particular, are needed. In this article, we describe and evaluate the statistical design of the AGILE platform, an adaptive randomized seamless Phase I/II trial platform ...

In the search for effective treatments for COVID-19, the initial emphasis has been on re-purposed treatments. To maximize the chances of finding successful treatments, novel treatments that have been developed for this disease in particular, are needed. In this article, we describe and evaluate the statistical design of the AGILE platform, an adaptive randomized seamless Phase I/II trial platform that seeks to quickly establish a safe range of doses and investigates treatments for potential efficacy. The bespoke Bayesian design (i) utilizes randomization during dose-finding, (ii) shares control arm information across the platform, and (iii) uses a time-to-event endpoint with a formal testing structure and error control for evaluation of potential efficacy. Both single-agent and combination treatments are considered. We find that the design can identify potential treatments that are safe and efficacious reliably with small to moderate sample sizes.



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Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftStatistical Methods in Medical Research
Verlag:Sage
Band:33
Nummer des Zeitschriftenheftes oder des Kapitels:11-12
Seitenbereich:S. 2115-2130
Datum14 Oktober 2024
InstitutionenInformatik und Data Science > Fachbereich Maschinelles Lernen und Data Science > Chair for Computational Statistics (Prof. Dr. Thomas Jaki)
Identifikationsnummer
WertTyp
10.1177/09622802241288348DOI
Stichwörter / KeywordsAdaptive platform trial, dose-escalation, COVID-19, randomized, seamless, time-to-improvement
Dewey-Dezimal-Klassifikation000 Informatik, Informationswissenschaft, allgemeine Werke > 004 Informatik
StatusVeröffentlicht
BegutachtetJa, diese Version wurde begutachtet
An der Universität Regensburg entstandenZum Teil
URN der UB Regensburgurn:nbn:de:bvb:355-epub-777674
Dokumenten-ID77767

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