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PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer

BACKGROUND: Personalized and effective treatments for pancreatic ductal adenocarcinoma (PDAC) continue to remain elusive. Novel clinical trial designs that enable continual and rapid evaluation of novel therapeutics are needed. Here, we describe a platform clinical trial to address this unmet need....

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Autores principales: Douglas, Julia E., Liu, Suyu, Ma, Junsheng, Wolff, Robert A., Pant, Shubham, Maitra, Anirban, Tamm, Eric P., Bhosale, Priya, Katz, Matthew H. G., Varadhachary, Gauri R., Koay, Eugene J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8722115/
https://www.ncbi.nlm.nih.gov/pubmed/34980020
http://dx.doi.org/10.1186/s12885-021-09095-7
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author Douglas, Julia E.
Liu, Suyu
Ma, Junsheng
Wolff, Robert A.
Pant, Shubham
Maitra, Anirban
Tamm, Eric P.
Bhosale, Priya
Katz, Matthew H. G.
Varadhachary, Gauri R.
Koay, Eugene J.
author_facet Douglas, Julia E.
Liu, Suyu
Ma, Junsheng
Wolff, Robert A.
Pant, Shubham
Maitra, Anirban
Tamm, Eric P.
Bhosale, Priya
Katz, Matthew H. G.
Varadhachary, Gauri R.
Koay, Eugene J.
author_sort Douglas, Julia E.
collection PubMed
description BACKGROUND: Personalized and effective treatments for pancreatic ductal adenocarcinoma (PDAC) continue to remain elusive. Novel clinical trial designs that enable continual and rapid evaluation of novel therapeutics are needed. Here, we describe a platform clinical trial to address this unmet need. METHODS: This is a phase II study using a Bayesian platform design to evaluate multiple experimental arms against a control arm in patients with PDAC. We first separate patients into three clinical stage groups of localized PDAC (resectable, borderline resectable, and locally advanced disease), and further divide each stage group based on treatment history (treatment naïve or previously treated). The clinical stage and treatment history therefore define 6 different cohorts, and each cohort has one control arm but may have one or more experimental arms running simultaneously. Within each cohort, adaptive randomization rules are applied and patients will be randomized to either an experimental arm or the control arm accordingly. The experimental arm(s) of each cohort are only compared to the applicable cohort specific control arm. Experimental arms may be added independently to one or more cohorts during the study. Multiple correlative studies for tissue, blood, and imaging are also incorporated. DISCUSSION: To date, PDAC has been treated as a single disease, despite knowledge that there is substantial heterogeneity in disease presentation and biology. It is recognized that the current approach of single arm phase II trials and traditional phase III randomized studies are not well-suited for more personalized treatment strategies in PDAC. The PIONEER Panc platform clinical trial is designed to overcome these challenges and help advance our treatment strategies for this deadly disease. TRIAL REGISTRATION: This study is approved by the Institutional Review Board (IRB) of MD Anderson Cancer Center, IRB-approved protocol 2020-0075. The PIONEER trial is registered at the US National Institutes of Health (ClinicalTrials.gov) NCT04481204. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s12885-021-09095-7.
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spelling pubmed-87221152022-01-06 PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer Douglas, Julia E. Liu, Suyu Ma, Junsheng Wolff, Robert A. Pant, Shubham Maitra, Anirban Tamm, Eric P. Bhosale, Priya Katz, Matthew H. G. Varadhachary, Gauri R. Koay, Eugene J. BMC Cancer Study Protocol BACKGROUND: Personalized and effective treatments for pancreatic ductal adenocarcinoma (PDAC) continue to remain elusive. Novel clinical trial designs that enable continual and rapid evaluation of novel therapeutics are needed. Here, we describe a platform clinical trial to address this unmet need. METHODS: This is a phase II study using a Bayesian platform design to evaluate multiple experimental arms against a control arm in patients with PDAC. We first separate patients into three clinical stage groups of localized PDAC (resectable, borderline resectable, and locally advanced disease), and further divide each stage group based on treatment history (treatment naïve or previously treated). The clinical stage and treatment history therefore define 6 different cohorts, and each cohort has one control arm but may have one or more experimental arms running simultaneously. Within each cohort, adaptive randomization rules are applied and patients will be randomized to either an experimental arm or the control arm accordingly. The experimental arm(s) of each cohort are only compared to the applicable cohort specific control arm. Experimental arms may be added independently to one or more cohorts during the study. Multiple correlative studies for tissue, blood, and imaging are also incorporated. DISCUSSION: To date, PDAC has been treated as a single disease, despite knowledge that there is substantial heterogeneity in disease presentation and biology. It is recognized that the current approach of single arm phase II trials and traditional phase III randomized studies are not well-suited for more personalized treatment strategies in PDAC. The PIONEER Panc platform clinical trial is designed to overcome these challenges and help advance our treatment strategies for this deadly disease. TRIAL REGISTRATION: This study is approved by the Institutional Review Board (IRB) of MD Anderson Cancer Center, IRB-approved protocol 2020-0075. The PIONEER trial is registered at the US National Institutes of Health (ClinicalTrials.gov) NCT04481204. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s12885-021-09095-7. BioMed Central 2022-01-03 /pmc/articles/PMC8722115/ /pubmed/34980020 http://dx.doi.org/10.1186/s12885-021-09095-7 Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Study Protocol
Douglas, Julia E.
Liu, Suyu
Ma, Junsheng
Wolff, Robert A.
Pant, Shubham
Maitra, Anirban
Tamm, Eric P.
Bhosale, Priya
Katz, Matthew H. G.
Varadhachary, Gauri R.
Koay, Eugene J.
PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer
title PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer
title_full PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer
title_fullStr PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer
title_full_unstemmed PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer
title_short PIONEER-Panc: a platform trial for phase II randomized investigations of new and emerging therapies for localized pancreatic cancer
title_sort pioneer-panc: a platform trial for phase ii randomized investigations of new and emerging therapies for localized pancreatic cancer
topic Study Protocol
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8722115/
https://www.ncbi.nlm.nih.gov/pubmed/34980020
http://dx.doi.org/10.1186/s12885-021-09095-7
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