Leflunomide for the treatment of patients with MEN1 syndrome
Descrizione riassuntiva dello studio
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)
Intervento studiato
Treatment with leflunomide 20 mg once daily for 6 months
(BASEC)
Malattie studiate
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)
Criteri di esclusione
- uncontrolled arterial hypertension - impaired kidney or liver function - active hepatitis B or C or severe immunosuppression, e.g. AIDS (BASEC)
Luogo dello studio
Basilea
(BASEC)
Sponsor
Julie Refardt
(BASEC)
Contatto per ulteriori informazioni sullo studio
Persona di contatto in Svizzera
Emanuel Christ
+41 61 265 25 25
emanuel.christ@clutterusb.chUniversity Hospital Basel
(BASEC)
Nome del comitato etico approvante (per studi multicentrici solo il comitato principale)
Commissione d'etica svizzera nord-ovest/centrale EKNZ
(BASEC)
Data di approvazione del comitato etico
08.11.2022
(BASEC)
ID di studio ICTRP
NCT05605587 (ICTRP)
Titolo ufficiale (approvato dal comitato etico)
The LUMEN1-trial (BASEC)
Titolo accademico
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Titolo pubblico
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Malattie studiate
MEN1 Gene Mutation (ICTRP)
Intervento studiato
Drug: Leflunomide 20 mg (ICTRP)
Tipo di studio
Interventional (ICTRP)
Disegno dello studio
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Criteri di inclusione/esclusione
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)
non disponibile
Endpoint primari e secondari
The primary outcome is the effect of a 6 months' treatment with leflunomide 20mg/day on MEN1-associated functional and non-functional tumors (ICTRP)
non disponibile
Data di registrazione
non disponibile
Inclusione del primo partecipante
non disponibile
Sponsor secondari
non disponibile
Contatti aggiuntivi
Julie Refardt, MD;Julie Refardt, julie.refardt@usb.ch, +41615565618;+41615565618 (ICTRP)
ID secondari
LUMEN1 (ICTRP)
Risultati-Dati individuali dei partecipanti
non disponibile
Ulteriori informazioni sullo studio
https://clinicaltrials.gov/ct2/show/NCT05605587 (ICTRP)
Risultati dello studio
Riepilogo dei risultati
non disponibile
Link ai risultati nel registro primario
non disponibile