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

Julia E. Douglas, Suyu Liu, Junsheng Ma, Robert A. Wolff, Shubham Pant, Anirban Maitra, Eric P. Tamm, Priya Bhosale, Matthew H.G. Katz, Gauri R. Varadhachary, Eugene J. Koay

Research output: Contribution to journalArticlepeer-review

7 Scopus citations


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 ( NCT04481204.

Original languageEnglish (US)
Article number14
Pages (from-to)14
JournalBMC Cancer
Issue number1
StatePublished - Jan 3 2022


  • Borderline resectable
  • Gemcitabine/nab-paclitaxel
  • Locally advanced
  • Pancreatic cancer
  • Pancreatic ductal adenocarcinoma (PDAC)
  • Phase II
  • Randomized platform trial
  • Resectable
  • Clinical Trials, Phase II as Topic/methods
  • Pancreatic Neoplasms/therapy
  • Humans
  • Randomized Controlled Trials as Topic/methods
  • Antineoplastic Protocols
  • Male
  • Treatment Outcome
  • Carcinoma, Pancreatic Ductal/therapy
  • Bayes Theorem
  • Adult
  • Female
  • Neoadjuvant Therapy/methods

ASJC Scopus subject areas

  • Genetics
  • Oncology
  • Cancer Research


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