Leflunomide for the treatment of patients with MEN1 syndrome
Résumé de l'étude
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 étudiée
Treatment with leflunomide 20 mg once daily for 6 months
(BASEC)
Maladie en cours d'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)
Critères d'exclusion
- uncontrolled arterial hypertension - impaired kidney or liver function - active hepatitis B or C or severe immunosuppression, e.g. AIDS (BASEC)
Lieu de l’étude
Bâle
(BASEC)
Sponsor
Julie Refardt
(BASEC)
Contact pour plus d'informations sur l'étude
Personne de contact en Suisse
Emanuel Christ
+41 61 265 25 25
emanuel.christ@clutterusb.chUniversity Hospital Basel
(BASEC)
Informations générales
+41 61 32 85522+41 61 32 85522
emanuel.christ@clutterusb.chemanuel.christ@clutterusb.ch(ICTRP)
Nom du comité d'éthique approbateur (pour les études multicentriques, uniquement le comité principal)
Ethikkommission Nordwest- und Zentralschweiz EKNZ
(BASEC)
Date d'approbation du comité d'éthique
08.11.2022
(BASEC)
Identifiant de l'essai ICTRP
NCT05605587 (ICTRP)
Titre officiel (approuvé par le comité d'éthique)
The LUMEN1-trial (BASEC)
Titre académique
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Titre public
Leflunomide Treatment for MEN1 Patients - the LUMEN1 Trial (ICTRP)
Maladie en cours d'investigation
MEN1 Gene Mutation (ICTRP)
Intervention étudiée
Drug: Leflunomide 20 mg (ICTRP)
Type d'essai
Interventional (ICTRP)
Plan de l'étude
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Critères d'inclusion/exclusion
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 disponible
Critères d'évaluation principaux et secondaires
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 disponible
Date d'enregistrement
non disponible
Inclusion du premier participant
non disponible
Sponsors secondaires
non disponible
Contacts supplémentaires
Julie Refardt, MD;Julie Refardt, julie.refardt@usb.ch, +41615565618;+41615565618 (ICTRP)
ID secondaires
LUMEN1 (ICTRP)
Résultats-Données individuelles des participants
non disponible
Informations complémentaires sur l'essai
https://clinicaltrials.gov/study/NCT05605587 (ICTRP)
Résultats de l'essai
Résumé des résultats
non disponible
Lien vers les résultats dans le registre primaire
non disponible