The effectiveness of Octreotide in hereditary hemorrhagic telangiectasia (a.k.a. Rendu-Osler-Weber disease) patients who suffer from gastrointestinal bleeding.
- Conditions
- Hereditary hemorrhagic telangiectasia (HHT)Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2018-004179-11-FR
- Lead Sponsor
- Radboudumc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 38
-Patients older than 18 years with written informed consent.
-Diagnosis of HHT: either confirmed by genetic testing or the Curaçao criteria (definite diagnosis) (7).
-Presence of 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, or other endoscopic treatment modality performed in the past 5 years.
-Diagnosis of iron deficiency anemia (IDA). IDA is defined as:
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 and
o The absence of other common causes of anemia (Vitamin B12 and/or folate deficiency in patients with macrocytic anemia and bone marrow disorders in patients with pancytopenia)
-Substantial transfusion dependency: at least 4 blood units and / or intravenous iron in the 6 months prior to study 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 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8
-Liver cirrhosis Child-Pugh C,
- Insulinoma,
- Uncontrolled diabetes mellitus as defined by HbA1c >64 mmol/ml, despite adequate therapy,
- Juvenile Polyposis Syndrome
- Symptomatic cholecystolithiasis (possible side-effect octreotide)
-Chronic or acute pancreatitis
-Bradycardia (heart rate below 50)*
-Hypersensitivity to the active ingredient (octreotide) or to auxiliary materials of the study medication
-Severe disease/comorbidities with a life expectancy < 1 year
-Use of chemotherapy and / or ciclosporin
-Pregnancy or nursing women or women who have a pregnancy wish during the study period.
-Women of childbearing potential who do not have a confirmed menstrual period and a negative, highly sensitive urine or serum pregnancy test < 7 days before inclusion
-Women of childbearing potential who do not use a highly effective contraceptive measure (according to section 4.1 of the Recommendations related to contraception and pregnancy testing in clinical trials of the Heads of Medicines Agencies (HMA)) or refuse to maintain this measure during the entire treatment- and relevant systemic exposure period.(25)
-Women of childbearing potential who refuse to undergo additional pregnancy testing during the treatment period
-Use of other anti-angiogenic drug treatment (thalidomide and/or bevacizumab)
-Use of hormonal (estrogen) therapy and/or antifibrinolytic therapy (tranexamic acid) to treat anemia due to telangiectasia with sufficient effect. However, when patients are red blood cell transfusion or iron infusion dependent despite these treatment options and are naïve to octreotide treatment, patients are still eligible for inclusion.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method