Pancreatic mucinous cystic neoplasm (MCN) is a precursor to invasive pancreatic adenocarcinoma which occurs almost exclusively in females in their 5th-7th decade. Currently the only option for MCN treatment and prevention of invasive pancreatic ductal adenocarcinoma (PDA) is oncologic resection. The clinical features of pancreatic MCN support the influence of sex hormones in the pathogenesis of the disease. Anti-hormonal therapy may therefore constitute an effective approach to treatment. Preliminary analyses from preclinical studies suggest that tamoxifen inhibits the spread and normal life cycle in MCN epithelial cells and fibroblasts. Investigators hypothesize that in humans, treatment with tamoxifen will lead to cyst regression or stabilization and may spare or delay the need for resection. Up to 15 participants not undergoing immediate resection will be enrolled and take tamoxifen orally for up to 24 weeks. The study will assess the feasibility of tamoxifen as a treatment for pancreatic MCN.
Pancreatic mucinous cystic neoplasm (MCN) is a precursor to invasive pancreatic adenocarcinoma which occurs almost exclusively in females in their 5th-7th decade. Currently the only option for MCN treatment and prevention of invasive pancreatic ductal adenocarcinoma (PDA) is oncologic resection. The clinical features of pancreatic MCN support the influence of sex hormones in the pathogenesis of the disease. Anti-hormonal therapy may therefore constitute an effective approach to treatment. Preliminary analyses from pre-clinical studies suggest that tamoxifen inhibits proliferation and apoptosis in MCN epithelial cells and fibroblasts. Investigators hypothesize that in humans, treatment with tamoxifen will lead to cyst regression or stabilization and may spare or delay the need for resection. This is an open-label pilot study of tamoxifen as chemoprevention in participants with pancreatic mucinous cystic neoplasms (MCN) who will not undergo immediate resection. Up to 15 participants will be enrolled and take tamoxifen 20mg by mouth daily for up to 24 weeks. The study will assess the feasibility of tamoxifen as chemoprevention in participants with pancreatic MCN and objective response rate as assessed by magnetic resonance imaging (MRI). This study is intended to inform the design of a subsequent trial formally powered to detect the benefit of tamoxifen in treating pancreatic MCN.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
PREVENTION
Masking
NONE
Enrollment
15
Tamoxifen is a nonsteroidal antiestrogen for oral administration.
University of Nebraska Medical Center
Omaha, Nebraska, United States
RECRUITINGFeasibility, by adherence rate
Adherence rate, defined as the proportion of participants who take ≥70% of the prescribed tamoxifen doses with adherence at least 60%, meaning ≥60% of participants take ≥70% of the prescribed tamoxifen doses.
Time frame: 6 months (approximately 180 days)
Feasibility, by retention rate
Retention rate, defined as the proportion of participants who complete imaging and study procedures at the 6-month visit with retention of at least 50%, meaning at least 50% of participants complete imaging and study procedures at the 6-month visit.
Time frame: 6 months (approximately 180 days)
Objective response rate (ORR) of pancreatic MCN
The ORR will be estimated based on the proportion of participants with complete or partial response on MRI/MRCP; partial response is defined by ≥ 20% decrease in maximal cyst diameter.
Time frame: 12 months (approximately 360 days)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.