Leflunomide treatment for patients with MEN1 gene mutation
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial
This study is testing if Leflunomide can help adults with MEN1 gene mutations manage their tumors and hormone issues better.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | University Hospital, Basel, Switzerland Academic / other |
| Locations | 1 site (Basel) |
| Trial ID | NCT05605587 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the use of Leflunomide, a medication traditionally used for rheumatoid arthritis, in treating patients with multiple endocrine neoplasia type 1 (MEN1) caused by mutations in the MEN1 gene. The study aims to assess the effectiveness of Leflunomide in managing associated tumors and hormonal syndromes in adult patients with confirmed MEN1 mutations. Participants will receive a daily dose of 20 mg of Leflunomide, and the trial will monitor their health outcomes over time. The goal is to explore a potential prophylactic treatment option for this hereditary condition, which currently lacks effective preventive measures.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with a known pathogenic MEN1 germline mutation and at least one associated tumor lesion or hormonal syndrome.
Not a fit: Patients with uncontrolled hypertension, impaired kidney or liver function, or cytopenia may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management and outcomes for patients with MEN1 by potentially preventing tumor development.
How similar studies have performed: While Leflunomide has been used in other contexts, this specific application for MEN1 is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Adult (≥18 years) patients with known pathogenic or likely pathogenic MEN1-germline mutation and at least 1 associated tumor lesion OR hormonal syndrome Exclusion Criteria: * uncontrolled arterial hypertension, defined as blood pressure \>160/100 mmHg * Impaired kidney function, defined as creatinine clearance \<50ml/min * Impaired liver function, defined as bilirubin or liver transaminases \>3 times upper normal range * Cytopenia, defined as one or several of the following: hemogloin \<100 g/l, leucopenia \<2x109/l, thrombocytopenia \<100x109/l
Where this trial is running
Basel
- Unispital Basel — Basel, Switzerland (Recruiting)
Study contacts
- Study coordinator: Julie Refardt, MD
- Email: julie.refardt@usb.ch
- Phone: +41615565618
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.