PAN1: A randomized phase II study evaluating potential predictive biomarkers in the treatment of metastatic pancreatic cancer.

Abstract

TPS4137 Background: Biomarker-based selection of cancer treatments has already entered routine clinical practice in some cancer types. Recent retrospective data suggests the human equilibrative nucleoside transporter 1 (hENT1) to be promising predictive marker for benefit from gemcitabine in pancreatic cancer, at least in resected disease. Methods: 80 patients will be randomised to receive either gemcitabine or FOLFOX chemotherapy (folinic acid (FOL) fluorouracil (F) and oxalipatin (OX)) with stratification based on hENT1 status. All patients will be required to have tumour samples tested for hENT1 prior to randomisation, with both hENT1 positive and negative patients eligible for inclusion. Patients will continue their assigned treatment until progression, or unacceptable toxicity. Primary endpoint: Progression free survival in each chemotherapy arm (FOLFOX/gemcitabine). Secondary endpoints: efficacy/activity of gemcitabine and FOLFOX as assessed by OS (overall survival), TTP (time to progression), RR (response rate according to RECIST v1.1), CA19.9 response; Percentage of eligible patients able to be randomised within 14 days of screening; PFS in each biomarker-selected cohort (hENT1 positive/negative); Treatment related toxicity in each chemotherapy group (CTCAE v4.0) and in biomarker cohorts (hENT1 positive/negative); establishment of a tissue bank from patients treated with and without gemcitabine for further biomarker studies. Eligibility: Adult patients with radiologically and histologically confirmed metastatic pancreatic adenocarcinoma who have not previously received treatment for metastatic disease, and who have tumour tissue available for hENT1 testing are eligible for the study. Patients who do not have tumour tissue available will be required to undergo core biopsy to obtain tissue for hENT1 testing. Status: Opened to accrual July 2011.