A Pilot Study of Chemoprevention With Tamoxifen in Patients With Pre-Invasive Pancreas Mucinous Cystic Neoplasms Who Will Not Undergo Immediate Resection
Categories (click each to see list of all clinical trials associated with that category): GI (ONC), Pancreatic Cancer Center of Excellence - PCCE (ONC)
Current Status: Open
Phase: Pilot (Cancer Prevention)
Principal Investigator: Klute, Kelsey
Contact Information:
Mahdi Hassan
mhassan@unmc.edu
Summary
Primary Objectives
1. To establish the feasibility of oral tamoxifen as chemoprevention in patients with pancreatic MCN and to inform
the design of a phase 2 clinical trial powered to assess response
2. To estimate the objective response rate (ORR) in pancreatic MCN treated with tamoxifen.
Secondary Objectives
To estimate the disease control rate
To characterize response dynamics
To explore the safety and toxicity of tamoxifen in the study population
To estimate the rate of cyst progression, including increase in maximal cyst diameter on magnetic resonance
imaging/magnetic resonance cholangiopancreatography (MRI/MRCP) by ≥ 20%, development of high-risk features
including solid component or mural nodule, main pancreatic ductal dilation or abrupt caliber change, obstructive
jaundice, lymphadenopathy; development of high-grade dysplasia or invasive PDA
Exploratory Objectives
To explore pharmacogenomic frequencies (CYP2D6 and CYP3A4 polymorphisms), concomitant medications
(inhibitors of CYP2D6 and CYP3A4), and metabolite levels (tamoxifen, endoxifen, N-desmethyltamoxifen and
4-hydroxytamoxifen levels) and their interaction with cyst response
To explore cyst outcomes following 24 weeks of treatment with tamoxifen
To explore the effect of tamoxifen on cyst fluid carcinoembryonic antigen (CEA), amylase levels
To explore the effect of tamoxifen on cyst epithelium and stromal proliferation and apoptosis
To explore blood biomarkers which may identify occult PDA or MCN at high risk of progression