Multiple Endocrine Neoplasia Type 1 (MEN1) Clinical Trials

Multiple endocrine neoplasia type 1 (MEN1), also called Wermer syndrome, is an autosomal dominant cancer predisposition syndrome caused by loss-of-function mutations in the MEN1 gene (chromosome 12q13) encoding menin, a nuclear tumor suppressor protein. Patients develop tumors in multiple endocrine and non-endocrine tissues with tumors typically arising in specific sequence by age. The parathyroid glands are involved in >95% of patients, usually the first manifestation (typically by age 20-25), causing primary hyperparathyroidism with hypercalcemia (often 11-13 mg/dL), nephrolithiasis (occurs in 20-40% from hypercalciuria), osteoporosis with increased fracture risk, and neuropsychiatric symptoms.

The most common symptoms of Multiple Endocrine Neoplasia Type 1 include Primary hyperparathyroidism with hypercalcemia, Gastroenteropancreatic neuroendocrine tumors, Pituitary adenomas, often prolactin-secreting, Nephrolithiasis from hypercalcemia, Carcinoid syndrome from neuroendocrine tumors, among others. Multiple Endocrine Neoplasia Type 1 affects approximately 1 in 20,000 to 30,000 people. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Multiple Endocrine Neoplasia Type 1 from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Clinical trials are investigating surveillance strategies to detect tumors earlier and targeted therapies including somatostatin analogs, mTOR inhibitors, and tyrosine kinase inhibitors for MEN1-associated tumors. Regular endocrine screening beginning in childhood (age 5-8) with biochemical testing, imaging, and genetic testing of family members enables early detection.

As of September 2026, Trial Friend's curated drug index does not include an FDA-approved treatment specific to Multiple Endocrine Neoplasia Type 1. The Treatments section of this page checks FDA drug labels live and lists any approved drug whose label names the condition, and the trials listed below cover the investigational options.

Multiple Endocrine Neoplasia Type 1 is caused by variants in the MEN1 gene. Germline MEN1 variants are usually inherited from an affected parent; tumors arise after a second, somatic hit in the other copy.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. The condition affects approximately 1 in 20,000 to 30,000 people. Genetic testing confirms the diagnosis.

Yes. Genetic testing for Multiple Endocrine Neoplasia Type 1 looks at the MEN1 gene. Germline MEN1 variants are usually inherited from an affected parent; tumors arise after a second, somatic hit in the other copy.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

A confirmed variant establishes the diagnosis and is often required to enroll in gene-specific trials. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Multiple Endocrine Neoplasia Type 1 clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Multiple Endocrine Neoplasia Type 1 clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.