Efficacy and safety of oral itraconazole in the reduction of epistaxis severity in hereditary hemorrhagic telangiectasia

Phase 2

Completed

Conditions: hereditary hemorrhagic telangiectasia
Rendu-Osler-Weber disease
10007510
10047043
10047075

Registration Number: NL-OMON46365

Lead Sponsor: Sint Antonius Ziekenhuis

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

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).

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 drugscontraindicated when using itraconazole (see protocol chapter 14.1 section G of the study protocol for a full list)
8. Patients who do not understand English or Dutch language sufficiently enough;
9. Patients who refuse informed consent.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary outcome of this study is the difference of epistaxis severity score<br /><br>between the baseline and at the end of the trial. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Safety of itraconazole, side-effects, adverse events<br /><br>Difference in monthly epistaxis severity measured with:<br /><br>- Monthly number of episodes;<br /><br>- Monthly duration;<br /><br>- Monthly intensity;<br /><br>- VAS score of epistaxis frequency, duration and intensity.<br /><br>Difference in biochemical blood values: Hb and ferritin between baseline and<br /><br>the end of the study.<br /><br>Difference in quality of life with SF-36 between baseline and end of the study</p><br>