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

NCT06320990 | Recruiting Phase 1 FDA Drug

• 1 other identifier: 0074-24-CB
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Pancreatic Cyst; Pancreatic Mucinous Cystic Neoplasm

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (1)

• Objective response rate (ORR) of pancreatic MCN

  12 months (approximately 360 days)

Study Arms (1)

Tamoxifen

EXPERIMENTAL

Tamoxifen 20mg by mouth daily for up to 6 months

Drug: Tamoxifen 20mg

Interventions

Tamoxifen 20mg DRUG

Tamoxifen is a nonsteroidal antiestrogen for oral administration.

Also known as: Soltamox

Tamoxifen

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Nebraska: lead

Study Sites (1)

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

RECRUITING

MeSH Terms

Conditions

Pancreatic Cyst

Interventions

Tamoxifen

Condition Hierarchy (Ancestors)

Cysts; Neoplasms; Pancreatic Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Stilbenes; Benzylidene Compounds; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals

Study Officials

• Kelsey Klute, MD

  University of Nebraska

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mahdi Hassan, MS

CONTACT

402-559-1947; mhassan@unmc.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 13, 2024
• First Posted: March 20, 2024
• Study Start: January 22, 2025
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): May 1, 2027
• Last Updated: June 8, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)