An uncontrolled, open label pilot-study assessing the efficacy in reducing bleeding severity, and the safety of oral tacrolimus in patients with hereditary hemorrhagic telangiectasia
- Conditions
- hereditary hemorrhagic telangiectasiaRendu-Osler-Weber syndrome10047075
- Registration Number
- NL-OMON48255
- Lead Sponsor
- Sint Antonius Ziekenhuis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 20
* Patients with HHT:
o Definite HHT according to the Curacao criteria (3 positive criteria or more)
AND/OR
o Genetically confirmed HHT
* Suffering from epistaxis at least on average of 4 days per week or documented
gastrointestinal teleangiectasis by endoscopy with suspicion of bleeding;
* In the last six months suffering from anemia, iron deficiency or use iron
treatment or blood transfusions;
* Failure or partial failure of local treatment with systemic treatment
indicated by ENT specialist or gastroenterologist;
* Adult (18 years or older at time of inclusion).
* Hypersensitivity or allergy for tacrolimus
* Patients with a severe disease with a life-expectancy <1 year;
* Women that are pregnant, nursing, have a pregnancy wish in the study period
or who use anticonception inadequately;
* Patients currently receiving chemotherapy;
* Severe kidney disease
* History of severe ventricular cardiac dysfunction
* A negative advise of a clinical pharmacist in cases with concurrent use of
drugs that have a clinically relevant interaction with tacrolimus.
* Patients who do not understand English or Dutch language sufficiently enough;
* Patients who refuse informed consent.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary study outcome is the difference in hemoglobin level at baseline<br /><br>compared to the level of hemoglobin after 20 weeks of treatment</p><br>
- Secondary Outcome Measures
Name Time Method