Leflunomide for the treatment of patients with MEN1 syndrome
Zusammenfassung der Studie
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)
Untersuchte Intervention
Treatment with leflunomide 20 mg once daily for 6 months
(BASEC)
Untersuchte Krankheit(en)
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)
Ausschlusskriterien
- uncontrolled arterial hypertension - impaired kidney or liver function - active hepatitis B or C or severe immunosuppression, e.g. AIDS (BASEC)
Studienstandort
Basel
(BASEC)
Sponsor
Julie Refardt
(BASEC)
Kontakt für weitere Auskünfte zur Studie
Kontaktperson Schweiz
Emanuel Christ
+41 61 265 25 25
emanuel.christ@clutterusb.chUniversity Hospital Basel
(BASEC)
Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)
Ethikkommission Nordwest- und Zentralschweiz EKNZ
(BASEC)
Datum der Bewilligung durch die Ethikkommission
08.11.2022
(BASEC)
ICTRP Studien-ID
NCT05605587 (ICTRP)
Offizieller Titel (Genehmigt von der Ethikkommission)
The LUMEN1-trial (BASEC)
Wissenschaftlicher Titel
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Öffentlicher Titel
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Untersuchte Krankheit(en)
MEN1 Gene Mutation (ICTRP)
Untersuchte Intervention
Drug: Leflunomide 20 mg (ICTRP)
Studientyp
Interventional (ICTRP)
Studiendesign
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Ein-/Ausschlusskriterien
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)
nicht verfügbar
Primäre und sekundäre Endpunkte
The primary outcome is the effect of a 6 months' treatment with leflunomide 20mg/day on MEN1-associated functional and non-functional tumors (ICTRP)
nicht verfügbar
Registrierungsdatum
nicht verfügbar
Einschluss des ersten Teilnehmers
nicht verfügbar
Sekundäre Sponsoren
nicht verfügbar
Weitere Kontakte
Julie Refardt, MD;Julie Refardt, julie.refardt@usb.ch, +41615565618;+41615565618 (ICTRP)
Sekundäre IDs
LUMEN1 (ICTRP)
Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
nicht verfügbar
Weitere Informationen zur Studie
https://clinicaltrials.gov/ct2/show/NCT05605587 (ICTRP)
Ergebnisse der Studie
Zusammenfassung der Ergebnisse
nicht verfügbar
Link zu den Ergebnissen im Primärregister
nicht verfügbar