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

Study Overview

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

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.

Pancreatic Cyst
Pancreatic Mucinous Cystic Neoplasm

DRUG: Tamoxifen 20mg

0074-24-CB

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates	Results Reporting Dates	Study Record Updates
First Submitted 2024-03-13	Results First Submitted N/A	Last Update Submitted that met QC Criteria 2025-06-04
First Submitted that Met QC Criteria 2024-03-13	Results First Submitted that Met QC Criteria N/A	Last Update Posted (ACTUAL) 2025-06-08
First Posted (ACTUAL) 2024-03-20	Results First Posted N/A	Last Verified 2025-06

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

Primary Purpose:
Prevention

Allocation:
Na

Interventional Model:
Single Group

Masking:
None

Arms and Interventions

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Tamoxifen Tamoxifen 20mg by mouth daily for up to 6 months	DRUG: Tamoxifen 20mg Tamoxifen is a nonsteroidal antiestrogen for oral administration.

Participant Group/Arm

Intervention/Treatment

EXPERIMENTAL: Tamoxifen

Tamoxifen 20mg by mouth daily for up to 6 months

DRUG: Tamoxifen 20mg

Tamoxifen is a nonsteroidal antiestrogen for oral administration.

Primary Outcome Measures	Measure Description	Time Frame
Feasibility, 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.	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.	6 months (approximately 180 days)

Secondary Outcome Measures	Measure Description	Time Frame
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.	12 months (approximately 360 days)

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Name: Mahdi Hassan, MS

Phone Number: 402-559-1947

Email: mhassan@unmc.edu

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person’s general health condition or prior treatments.

Ages Eligible for Study:
ALL

Sexes Eligible for Study:
19 Years

Accepts Healthy Volunteers:

Age ≥ 19 years
Clinically diagnosed pre-invasive pancreatic mucinous cystic neoplasm (MCN), measurable by cross-sectional imaging
Surgical resection of the lesion is not planned due to cyst features, patient factors or patient preference
Females of reproductive potential and males with partners of reproductive potential must agree to employ two methods contraception throughout the study and for up to 3 months following treatment. Non-child-bearing potential is defined as age 45 years or older and no menses for greater than or equal to 12 months or any age with surgical removal of the uterus and/or both ovaries.
Estimated glomerular filtration rate (eGFR) > 30mL/min/1.73m2
Willing and able to provide informed consent to and abide by the protocol

Presence of invasive pancreatic adenocarcinoma or high-grade dysplasia
Presence of a solid component or mural nodule, main pancreatic duct dilation or abrupt caliber change, obstructive jaundice, lymphadenopathy
Current or prior use of tamoxifen or another estrogen antagonist including, but not limited to, clomifene, raloxifene, fulvestrant, anastrazole; subjects who have previously used an estrogen antagonist are eligible provided the last use was at least 5 years prior to enrollment.
Current or planned use of hormonal treatments including estrogen, progesterone, androgens, hormone replacement therapy or other types of hormonal contraceptives including implants and depot injections; levonorgestrel-releasing intrauterine device (IUD) is permitted.
Contraindications to tamoxifen include:

Pregnancy or nursing
Known allergy or hypersensitivity to tamoxifen
Cataracts which affect visual acuity (ie. symptomatic)
Retinopathy which affects visual acuity (ie. symptomatic)
Current warfarin use
History of deep vein thrombosis or pulmonary embolism or other condition which, in the opinion of the investigator, may significantly increase the individual's risk of venous thromboembolism
History of stroke
Known endometrial hyperplasia or personal history of endometrial carcinoma, uterine sarcoma and uterine carcinosarcoma
History of intestinal disease or major gastric surgery likely to alter absorption of tamoxifen or inability to swallow oral medications
Uncontrolled illness including but not limited to ongoing or active infection requiring IV antibiotics, symptomatic congestive heart failure, unstable angina or uncontrolled cardiac arrhythmias, or other conditions which might jeopardize or preclude the ability of the patient to take tamoxifen or the safety of follow-up visits, scans and procedures
Elective surgery planned for the study period
Participation in another clinical study with an investigational product during the last 28 days
Any participant, in the opinion of the investigator, who will not be able to tolerate treatment, or the participant is unsuitable to participate in the study and is unlikely to comply with study procedures, restrictions and requirements

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

PRINCIPAL_INVESTIGATOR: Kelsey Klute, MD, University of Nebraska

Publications

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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Clinical Trial Record