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

Efficacy and safety of oral itraconazole in the reduction of epistaxis severity in hereditary hemorrhagic telangiectasia - Itraconazole for epistaxis in HHT patients

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-003272-31-NL

Enrollment

Registered

2018-01-11

Start date

2018-05-18

Completion date

Unknown

Last updated

2018-06-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Severe epistaxis in patients with Hereditary Hemorrhagic Telangiectasia also known as Rendu-Osler-Weber disease MedDRA version: 20.0 Level: LLT Classification code 10031132 Term: Osler-Weber-Rendu disease System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10038554 Term: Rendu-Osler-Weber syndrome System Organ Class: 100000004850

Interventions

Trade Name: Sporanox Product Name: Itraconazole Pharmaceutical Form: Capsule

Eligibility

Sex/Gender

All

Inclusion criteria

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

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.

Design outcomes

Primary

Measure	Time frame
Main Objective: Study hypothesis: The primary study hypothesis states that oral treatment with itraconazole will reduce the epistaxis severity and frequency due to the anti-angiogenic effects of itraconazole. Study objective: To assess the efficacy and safety of oral itraconazole in HHT patients with severe epistaxis;Secondary Objective: Difference in monthly epistaxis duration and frequency after treatment compared to baseline. Difference in the mean hemoglobin (Hb) and ferritin levels in HHT patients with epistaxis after treatment compared to baseline. Difference in quality of life in HHT patients with epistaxis following treatment after treatment compared to baseline. Effects of itraconazole on other HHT associated symptoms such as gastro-intestinal blood loss and presence of telangiectasias after treatment compared to baseline. Safety, side-effects and (serious) adverse events of oral itraconazole in HHT patients with epistaxis. Long term efficacy of treatment at 1 and 3 months after itraconazole treatment has ended on the severity of epistaxis. ;Primary end point(s): The primary end point used in this study is the epistaxis severity score (ESS);Timepoint(s) of evaluation of this end point: End point will be evaluated after 16 weeks of treatment	—

Measure

Time frame

Main Objective: Study hypothesis: The primary study hypothesis states that oral treatment with itraconazole will reduce the epistaxis severity and frequency due to the anti-angiogenic effects of itraconazole. Study objective: To assess the efficacy and safety of oral itraconazole in HHT patients with severe epistaxis;Secondary Objective: Difference in monthly epistaxis duration and frequency after treatment compared to baseline. Difference in the mean hemoglobin (Hb) and ferritin levels in HHT patients with epistaxis after treatment compared to baseline. Difference in quality of life in HHT patients with epistaxis following treatment after treatment compared to baseline. Effects of itraconazole on other HHT associated symptoms such as gastro-intestinal blood loss and presence of telangiectasias after treatment compared to baseline. Safety, side-effects and (serious) adverse events of oral itraconazole in HHT patients with epistaxis. Long term efficacy of treatment at 1 and 3 months after itraconazole treatment has ended on the severity of epistaxis. ;Primary end point(s): The primary end point used in this study is the epistaxis severity score (ESS);Timepoint(s) of evaluation of this end point: End point will be evaluated after 16 weeks of treatment

—

Secondary

Measure	Time frame
Secondary end point(s): The secondary end points in this study are safety of therapy, the frequency and number of monthly epistaxis measured with an epistaxis diary, hemoglobin levels, ferritin levels, and the quality of life measured with the Short-Form Questionnaire 36 (SF-36).;Timepoint(s) of evaluation of this end point: End point will be evaluated after 16 weeks of treatment	—

Countries

Netherlands

Contacts

Public ContactCentral study coordinator

St. Antonius Ziekenhuis

s.kroon@antoniusziekenhuis.nl0031883201579

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026