Itraconazole as treatment for severe nose bleeding in patients with hereditary hemorrhagic telangiectasia

Phase 1

Conditions: Severe epistaxis in patients with Hereditary Hemorrhagic Telangiectasia also known as Rendu-Osler-Weber disease
MedDRA version: 20.0Level: LLTClassification code 10031132Term: Osler-Weber-Rendu diseaseSystem Organ Class: 100000004850
MedDRA version: 20.0Level: LLTClassification code 10038554Term: Rendu-Osler-Weber syndromeSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2017-003272-31-NL

Lead Sponsor: St. Antonius Ziekenhuis

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 25

Inclusion Criteria

1. Patients with HHT: Definite HHT according to the Curacao criteria (3 positive criteria or more) AND/OR genetically confirmed HHT
2. Suffering from epistaxis at least on average of 4 days per week;
3. In the last six months suffering from anemia or iron deficiency or in last twelve months use iron treatment or blood transfusions;
4. Failure or partial failure of local treatment with systemic treatment indicated by ENT specialist;
5. Adult (18 years or older at time of inclusion).

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 13

Exclusion Criteria

1. Patients with any history of ventricular cardiac dysfunction;
2. Patients with elevated liver enzymes or any pre-existing liver disease or a history with known liver toxicity caused by medication;
3. Hypersensitivity or allergy for azole antifungal drugs;
4. Patients with a severe disease with a life-expectancy <1 year;
5. Women that are pregnant, nursing, have a pregnancy wish in the study period or who use anticonception inadequately;
6. Patients currently receiving chemotherapy;
7. Patients receiving drugs that are metabolized by CYP3A: that prolongate QT-interval (mizolastine, pimozide, sertindole of quinidine), orally administered midazolam, statins (simvastatin, atorvastatin), alkaloids (ergotamine), eletriptan, or drugs that induce CYP3A4 (rifampicin, rifabutin, phenytoin, carbamazepine, phenobarbital or St John's wort (Hypericum perforatum));
8. Patients who do not understand English or Dutch language sufficiently enough;
9. Patients who refuse informed consent.