Leflunomide for the treatment of patients with MEN1 syndrome
Summary description of the study
Leflunomide has been used for many years to treat rheumatoid arthritis. Several studies in tumor cells and mice have shown that leflunomide has growth-inhibitory effects on various tumors. In a recent study, leflunomide specifically destroyed MEN1-deficient cells, prevented the onset of pancreatic tumors in mice, and led to tumor regression/stabilization in three MEN1 patients with advanced aggressive pancreatic tumors. Accordingly, leflunomide could be used as a new treatment option for patients with known MEN1 mutation. The aim of this study is therefore to investigate the antitumor effect of leflunomide treatment on MEN1-associated tumors in patients with known MEN1 syndrome.
(BASEC)
Intervention under investigation
Treatment with leflunomide 20 mg once daily for 6 months
(BASEC)
Disease under investigation
Multiple endocrine neoplasia type 1 (MEN1) is an autosomal dominant inherited disorder caused by mutations in the tumor suppressor gene MEN1 with the corresponding gene product Menin. MEN1 is characterized by the occurrence of parathyroid, pancreatic, and pituitary tumors that can release excessive amounts of hormones. To date, there is no prophylactic treatment to prevent tumor development in this hereditary disease.
(BASEC)
Adults (≥ 18 years) with MEN1 mutation and at least 1 associated tumor lesion OR hormonal syndrome (BASEC)
Exclusion criteria
- uncontrolled arterial hypertension - impaired kidney or liver function - active hepatitis B or C or severe immunosuppression, e.g. AIDS (BASEC)
Trial sites
Basel
(BASEC)
Sponsor
Julie Refardt
(BASEC)
Contact
Contact Person Switzerland
Emanuel Christ
+41 61 265 25 25
emanuel.christ@clutterusb.chUniversity Hospital Basel
(BASEC)
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee northwest/central Switzerland EKNZ
(BASEC)
Date of authorisation
08.11.2022
(BASEC)
ICTRP Trial ID
NCT05605587 (ICTRP)
Official title (approved by ethics committee)
The LUMEN1-trial (BASEC)
Academic title
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Public title
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Disease under investigation
MEN1 Gene Mutation (ICTRP)
Intervention under investigation
Drug: Leflunomide 20 mg (ICTRP)
Type of trial
Interventional (ICTRP)
Trial design
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Inclusion/Exclusion criteria
Gender: All
Maximum age: N/A
Minimum age: 18 Years
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 (ICTRP)
not available
Primary and secondary end points
The primary outcome is the effect of a 6 months' treatment with leflunomide 20mg/day on MEN1-associated functional and non-functional tumors (ICTRP)
not available
Registration date
not available
Incorporation of the first participant
not available
Secondary sponsors
not available
Additional contacts
Julie Refardt, MD;Julie Refardt, julie.refardt@usb.ch, +41615565618;+41615565618 (ICTRP)
Secondary trial IDs
LUMEN1 (ICTRP)
Results-Individual Participant Data (IPD)
not available
Further information on the trial
https://clinicaltrials.gov/ct2/show/NCT05605587 (ICTRP)
Results of the trial
Results summary
not available
Link to the results in the primary register
not available