Tamoxifen for Pancreatic Cysts

(MCN_Tam Trial)

Overseen By Mahdi Hassan, MS

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of Nebraska

Must not be taking: Estrogen antagonists, Hormonal treatments

Disqualifiers: Invasive adenocarcinoma, Stroke, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether tamoxifen, a medication typically used for breast cancer, can help with pancreatic mucinous cystic neoplasm (MCN), a type of pancreatic cyst. Researchers believe tamoxifen may shrink the cysts or halt their growth, potentially eliminating the need for surgery. The trial seeks participants diagnosed with MCN who do not plan to undergo surgery immediately. As a Phase 1 trial, the research focuses on understanding how tamoxifen affects people with MCN, offering participants a chance to explore this potential treatment.

Will I have to stop taking my current medications?

The trial requires that you stop using tamoxifen or other estrogen antagonists, as well as certain hormonal treatments like estrogen, progesterone, and androgens. If you are currently using warfarin, you will also need to stop. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Studies have shown that tamoxifen is generally well-tolerated, but some possible side effects require attention. In certain cases, tamoxifen has been linked to pancreatitis, an inflammation of the pancreas, usually occurring with very high levels of triglycerides, a type of fat in the blood. Reports have also noted liver cysts and very rare cases of endometrial cancer, which affects the lining of the uterus.

While these side effects can sound serious, they do not occur in everyone. Many people take tamoxifen without major problems. This study, in its early phase, aims to gather more information on safety and how well participants tolerate the treatment. Participants in the trial will have their health closely monitored for any side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for pancreatic cysts, which often involve surgical removal or close monitoring, Tamoxifen offers a non-invasive and potentially more manageable option. Tamoxifen is traditionally used in breast cancer treatment and works by blocking estrogen receptors, which might play a role in cyst development. Researchers are excited because it represents a novel approach that could reduce the need for surgery and offer a simpler, oral medication route for managing pancreatic cysts.

What evidence suggests that tamoxifen might be an effective treatment for pancreatic cysts?

Research suggests that tamoxifen, which participants in this trial will receive, could help treat pancreatic cysts by affecting the involved cells. Early studies indicate that tamoxifen can halt the growth and normal cycle of certain cells in these cysts. This might shrink or stabilize the cysts, possibly reducing the need for surgery. Other studies have shown that tamoxifen causes cell death in some cancer cells. While direct evidence in humans is limited, these findings support the potential of tamoxifen in managing pancreatic cysts. ^{1 3 6 7 8}

Who Is on the Research Team?

Kelsey Klute, MD

Principal Investigator

University of Nebraska

Are You a Good Fit for This Trial?

This trial is for individuals with certain types of non-cancerous pancreatic cysts known as mucinous cystic neoplasms (MCNs) who are not scheduled for immediate surgery to remove these cysts. Specific eligibility details are not provided.

Inclusion Criteria

Willing and able to provide informed consent to and abide by the protocol

Estimated glomerular filtration rate (eGFR) > 30mL/min/1.73m2

My condition is a type of non-invasive pancreatic cyst.

Exclusion Criteria

I have been diagnosed with a serious type of pancreatic cancer or pre-cancer.

I am using or plan to use hormonal treatments, but I can use a levonorgestrel-releasing IUD.

I do not have conditions that prevent me from taking tamoxifen.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive tamoxifen 20mg by mouth daily for up to 24 weeks

24 weeks

Regular visits for monitoring and adherence checks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

MRI/MRCP assessments to evaluate response

What Are the Treatments Tested in This Trial?

Interventions

• Tamoxifen

Trial Overview

The study is testing the effectiveness of a medication called Tamoxifen, taken orally at a dose of 20mg daily, in preventing cancer in patients with MCN. Up to 15 participants will be involved and the treatment duration is up to 24 weeks.

How Is the Trial Designed?

Group I: Tamoxifen Experimental Treatment 1 Intervention

Tamoxifen 20mg by mouth daily for up to 6 months

Tamoxifen is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Nolvadex for:

• Breast cancer
• Infertility
• Gynecomastia

Approved in United States as Tamoxifen citrate for:

• Breast cancer
• Reduction in breast cancer incidence in high-risk women
• McCune-Albright Syndrome

Approved in Canada as Tamoxifen for:

• Breast cancer
• Reduction in breast cancer incidence in high-risk women

Approved in Japan as Tamoxifen for:

• Breast cancer

Who Is Running the Clinical Trial?

University of Nebraska

Lead Sponsor

Trials

563

Recruited

1,147,000+

Published Research Related to This Trial

In a study of 65 patients with resected pancreatic adenocarcinoma, tamoxifen combined with immunochemotherapy significantly improved survival rates compared to those not receiving tamoxifen (p < 0.01).

Tamoxifen showed particularly strong benefits in male patients, with a one-year survival rate of 85.6% for those with carcinoma in the head of the pancreas, compared to only 19.1% in the non-tamoxifen group, highlighting its potential as an effective adjuvant therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The beneficial effect of tamoxifen therapy in patients with resected adenocarcinoma of the pancreas. Horimi, T., Takasaki, M., Toki, A., et al.[2013]

Tamoxifen therapy can lead to severe acute pancreatitis, as demonstrated by a case report of a 44-year-old woman who experienced recurrent episodes while on the medication.

Given the risks associated with tamoxifen-induced pancreatitis, clinicians should avoid rechallenging patients with tamoxifen if it is suspected to be the cause of pancreatitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Severe acute pancreatitis due to tamoxifen-induced hypertriglyceridemia with positive rechallenge. Sakhri, J., Ben Salem, C., Harbi, H., et al.[2013]

In a study of 22,005 breast cancer patients, tamoxifen use was not significantly associated with an increased risk of acute pancreatitis (AP), with an adjusted hazard ratio of 0.94, indicating no correlation.

The research found no dose-response relationship between tamoxifen use and AP risk, suggesting that tamoxifen is not a contributing factor to the development of AP in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tamoxifen use and acute pancreatitis: A population-based cohort study. Hsu, FG., Hsieh, YW., Sheu, MJ., et al.[2019]

Tamoxifen has shown promising results in phase II clinical trials for pancreatic cancer, with most trials resulting in at least double the average survival, suggesting its potential as an effective treatment despite its lack of effect in laboratory settings.

Cyproterone, an anti-androgen, inhibits tumor growth in pancreatic cancer, while tamoxifen may also serve as a targeted carrier for other cytotoxic drugs, highlighting the need for further investigation into its mechanisms and applications in human patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hormones in chemotherapy for pancreatic cancer, chemoagents or carriers? Siu, TO., Kwan, WB.[2013]

In a study involving 14 patients with unresectable pancreatic adenocarcinoma, treatment with tamoxifen did not show any objective response, indicating a lack of efficacy in this context.

Despite previous suggestions that tamoxifen could increase survival in pancreatic cancer patients, this study's results do not support that claim for endocrine manipulation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase II study to evaluate tamoxifen in pancreatic adenocarcinoma. Crowson, MC., Dorrell, A., Rolfe, EB., et al.[2013]

In a study involving 11 patients with unresectable pancreatic carcinoma, hormone therapy using tamoxifen showed a mean survival of 235 days in 4 treated patients, compared to only 128 days in 7 control patients.

Two patients receiving tamoxifen had particularly long survival times of 321 and 463 days, suggesting that tamoxifen may be an effective treatment option for this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Hormone therapy using tamoxifen in unresectable carcinoma of the pancreas--preliminary study]. Morita, S., Motohara, T., Ogawa, K., et al.[2013]

Tamoxifen, an anti-estrogen, has been tested in clinical trials for patients with unresectable pancreatic carcinoma, but its effectiveness as a single agent has shown uncertain results regarding survival benefits compared to untreated controls.

Combination treatments, such as intravenous Onconase with oral tamoxifen, may provide a significant survival advantage for some patients with advanced pancreatic cancer, suggesting that synergistic therapies could be more effective than single-agent treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tamoxifen in patients with advanced pancreatic adenocarcinoma. Mikulski, S.[2011]

In a randomized placebo-controlled trial involving 44 patients with irresectable adenocarcinoma of the pancreas, tamoxifen did not significantly improve survival compared to placebo.

Quality-of-life assessments also showed no significant differences between patients treated with tamoxifen and those receiving placebo, indicating that tamoxifen does not provide a meaningful benefit for this type of cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical trial of tamoxifen in patients with irresectable pancreatic adenocarcinoma. The Yorkshire Gastrointestinal Tumour Group. Taylor, OM., Benson, EA., McMahon, MJ.[2019]

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This is an open-label pilot study of tamoxifen as chemoprevention in participants with pancreatic mucinous cystic neoplasms (MCN) who will not undergo immediate ...