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A Seamless Phase I/II Platform Design with a Time-To-Event Efficacy Endpoint for Potential COVID-19 Therapies

Research output: Contribution to Journal/Magazine › Journal article › peer-review

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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 et al.
In: Statistical Methods in Medical Research, Vol. 33, No. 11-12, 30.11.2024, p. 2115-2130.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Harvard

Jaki, T, Barnett, H, Titman, A & Mozgunov, P 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, vol. 33, no. 11-12, pp. 2115-2130. https://doi.org/10.1177/09622802241288348

APA

Jaki, T., Barnett, H., Titman, A., & Mozgunov, P. (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), 2115-2130. https://doi.org/10.1177/09622802241288348

Vancouver

Jaki T, Barnett H, Titman A, Mozgunov P. A Seamless Phase I/II Platform Design with a Time-To-Event Efficacy Endpoint for Potential COVID-19 Therapies. Statistical Methods in Medical Research. 2024 Nov 30;33(11-12):2115-2130. Epub 2024 Oct 14. doi: 10.1177/09622802241288348

Author

Jaki, Thomas ; Barnett, Helen ; Titman, Andrew et al. / A Seamless Phase I/II Platform Design with a Time-To-Event Efficacy Endpoint for Potential COVID-19 Therapies. In: Statistical Methods in Medical Research. 2024 ; Vol. 33, No. 11-12. pp. 2115-2130.

Bibtex

@article{1490ad9ee7fc40df9cb1c8e20ce69fb6,
title = "A Seamless Phase I/II Platform Design with a Time-To-Event Efficacy Endpoint for Potential COVID-19 Therapies",
abstract = "In the search for effective treatments for COVID-19, initial emphasis has been on re-purposed treatments. To maximise the chances of finding successful treatments, novel treatments that have been developed for this disease in particular, are needed. In this manuscript 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. ",
author = "Thomas Jaki and Helen Barnett and Andrew Titman and Pavel Mozgunov",
year = "2024",
month = nov,
day = "30",
doi = "10.1177/09622802241288348",
language = "English",
volume = "33",
pages = "2115--2130",
journal = "Statistical Methods in Medical Research",
issn = "0962-2802",
publisher = "SAGE Publications Ltd",
number = "11-12",

}

RIS

TY - JOUR

T1 - A Seamless Phase I/II Platform Design with a Time-To-Event Efficacy Endpoint for Potential COVID-19 Therapies

AU - Jaki, Thomas

AU - Barnett, Helen

AU - Titman, Andrew

AU - Mozgunov, Pavel

PY - 2024/11/30

Y1 - 2024/11/30

N2 - In the search for effective treatments for COVID-19, initial emphasis has been on re-purposed treatments. To maximise the chances of finding successful treatments, novel treatments that have been developed for this disease in particular, are needed. In this manuscript 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.

AB - In the search for effective treatments for COVID-19, initial emphasis has been on re-purposed treatments. To maximise the chances of finding successful treatments, novel treatments that have been developed for this disease in particular, are needed. In this manuscript 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.

U2 - 10.1177/09622802241288348

DO - 10.1177/09622802241288348

M3 - Journal article

C2 - 39397762

VL - 33

SP - 2115

EP - 2130

JO - Statistical Methods in Medical Research

JF - Statistical Methods in Medical Research

SN - 0962-2802

IS - 11-12

ER -