NCT06320990

Chemoprevention With Tamoxifen in Pre-Invasive Pancreas Mucinous Cystic Neoplasms Not Undergoing Immediate Resection

A Pilot Study of Chemoprevention With Tamoxifen in Patients With Pre-Invasive Pancreas Mucinous Cystic Neoplasms Who Will Not Undergo Immediate Resection

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: University of Nebraska · Academic / Other
• Sex: All
• Age: 19 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Pancreatic Cyst
• Pancreatic Mucinous Cystic Neoplasm

Interventions

Type	Name	Description
DRUG	Tamoxifen 20mg	Tamoxifen is a nonsteroidal antiestrogen for oral administration.

Timeline

• Start date: 2025-01-22
• Primary completion: 2026-11-01
• Completion: 2027-05-01
• First posted: 2024-03-20
• Last updated: 2025-06-08

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study