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

BMC Cancer

Junsheng Ma¹  Suyu Liu¹  Eric P. Tamm²  Priya Bhosale²  Shubham Pant³  Robert A. Wolff³  Gauri R. Varadhachary³  Julia E. Douglas⁴  Eugene J. Koay⁴  Matthew H. G. Katz⁵  Anirban Maitra⁶ 

[1] Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA;Department of Diagnostic Radiology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA;Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA;Department of Radiation Oncology, The University of Texas MD Anderson Cancer Center, 1220 Holcombe Boulevard, MS97, 77030, Houston, TX, USA;Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA;Sheikh Ahmed Center for Pancreatic Cancer Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA;Department of Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA;

关键词: Pancreatic cancer;    PIONEER;    Phase II;    Randomized platform trial;    Resectable;    Borderline resectable;    Locally advanced;    FOLFIRINOX;    Gemcitabine/nab-paclitaxel;    Pancreatic ductal adenocarcinoma (PDAC);   

DOI  :  10.1186/s12885-021-09095-7

来源: Springer

【 摘 要 】

BackgroundPersonalized 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.MethodsThis 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.DiscussionTo 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 registrationThis 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.

【 授权许可】

CC BY