Using tamoxifen to prevent cancer in patients with pancreatic mucinous cystic neoplasms
A Pilot Study of Chemoprevention With Tamoxifen in Patients With Pre-Invasive Pancreas Mucinous Cystic Neoplasms Who Will Not Undergo Immediate Resection
This study is testing if taking tamoxifen can help people with pancreatic mucinous cystic neoplasms avoid surgery by shrinking or stabilizing their cysts.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 19 Years and up |
| Sex | All |
| Sponsor | University of Nebraska Academic / other |
| Locations | 1 site (Omaha, Nebraska) |
| Trial ID | NCT06320990 on ClinicalTrials.gov |
What this trial studies
This open-label pilot study investigates the use of tamoxifen as a chemopreventive agent in patients with pancreatic mucinous cystic neoplasms (MCN) who are not undergoing immediate surgical resection. Up to 15 participants will take tamoxifen 20mg daily for up to 24 weeks, with the primary endpoints focusing on the feasibility of this treatment and the objective response rate assessed through MRI. The study aims to gather preliminary data that could inform a larger, more definitive trial in the future. The hypothesis is that tamoxifen may lead to cyst regression or stabilization, potentially delaying the need for surgery.
Who should consider this trial
Good fit: Ideal candidates are adults aged 19 and older with clinically diagnosed pre-invasive pancreatic mucinous cystic neoplasms who are not candidates for immediate surgery.
Not a fit: Patients with invasive pancreatic adenocarcinoma or high-grade dysplasia will not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a non-surgical treatment option that delays or spares patients from the need for resection of pancreatic cysts.
How similar studies have performed: While this approach is novel in the context of pancreatic MCN, preliminary analyses from pre-clinical studies suggest potential efficacy of tamoxifen in similar scenarios.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * 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
Where this trial is running
Omaha, Nebraska
- University of Nebraska Medical Center — Omaha, Nebraska, United States (Recruiting)
Study contacts
- Principal investigator: Kelsey Klute, MD — University of Nebraska
- Study coordinator: Mahdi Hassan, MS
- Email: mhassan@unmc.edu
- Phone: 402-559-1947
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.