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The effectiveness of Octreotide in hereditary hemorrhagic telangiectasia (a.k.a. Rendu-Osler-Weber disease) patients who suffer from gastrointestinal bleeding.

Phase 1
Conditions
Hereditary hemorrhagic telangiectasia (HHT)
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Registration Number
EUCTR2018-004179-11-NL
Lead Sponsor
Radboudumc
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
Not specified
Target Recruitment
38
Inclusion Criteria

- Patients older than 18 years with written informed consent.
- Diagnosis of HHT: either confirmed by genetic testing or the Curacao criteria (definite
diagnosis).
- Presence of IDA in combination with the presence endoscopic proven GI AVM manifestations /
telangiectasias confirmed within the last 12 months (upper and/or lower endoscopy and/or
capsule endoscopy).
- Endoscopic refractory: at least 1 endoscopic APC / laser /other endoscopic treatment modality
performed in the past 5 years.
- Substantial transfusion dependency: at least 4 blood units and / or intravenous iron in the 6
months prior to study inclusion with a:
o At least one serum ferritin below < 30 ug/l within the last 6 months requiring iron
infusion above or equal to 1 g and/or
o Hemoglobin below 5.6 mmol/l (9.0 g/dl) or are in need of transfusions due to anemia related symptoms within the last 6 months requiring blood transfusion above.
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 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8

Exclusion Criteria

- liver cirrhosis child-pugh C.
- symptomatic cholecystolithiasis (possible side-effect octreotide).
- pregnancy or nursing women or women have a pregnancy wish during the study period.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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