Tacrolimus for bleeding in hereditary hemorrhagic telangiectasia patients

Phase 1

• Conditions: Gastrointestinal bleeding and epistaxis caused by hereditary hemorrhagic telangiectasia. Hereditary Hemorrhagic Telangiectasia (HHT) is an autosomal dominant inherited disease characterized by mucocutaneous telangiectasis. Telangiectasis predominantly observed in the nasal mucosa and gut, and are abnormal, thin walled blood vessel that can easily rupture leading to hemorrhage.; MedDRA version: 20.0 Level: LLT Classification code 10038554 Term: Rendu-Osler-Weber syndrome System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-003585-40-NL
• Lead Sponsor: St. Antonius Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-09-30
• Last Update Posted: 1 February 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 20

Inclusion Criteria

•Patients with HHT:
oDefinite HHT according to the Curacao criteria (3 positive criteria or more)
AND/OR
oGenetically 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).

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 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

•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;
•Patients receiving drugs that are contraindicated when using tacrolimus (see chapter 14.1, section G).
•Patients who do not understand English or Dutch language sufficiently enough;
•Patients who refuse informed consent.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified