💬In plain words
What the trial is, and whether it could suit you
Patients affected by hereditary hemorrhagic telangiectasia (HHT) very often suffer from recurrent nosebleeds called epistaxis. There is no treatment currently available to reduce the frequency or severity of epistaxis.
This research project will examine the effect of nintedanib, a capsule to be taken twice a day, on the frequency and severity of epistaxis in HHT.
The study will take place at the Respiratory medicine department of the Lausanne University Hospital (Centre hospitalier universitaire vaudois, CHUV). The investigators will recruit about 48 participants with HHT, who will be divided in 2 groups. Each group will perform the same examinations and follow-up visits. The study will begin with 2 months of observation during which subjects will be asked to fill a diary to record the number and duration of epistaxis episodes. The diary will be filled daily for the entire duration of
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
Inclusion criteria:
1. signed informed consent
2. definite HHT disease (defined as the presence of a pathogenic mutation in one of the HHT genes, or the presence of 3 out of 4 Curaçao clinical criteria)
3. age ≥18 years at the time of informed consent
4. moderate to serious epistaxis defined as Epistaxis Severity Score (ESS) ≥2.5
5. absence of cerebral arteriovenous malformation demonstrated by brain imaging
Exclusion criteria:
1. Women who are pregnant or breastfeeding
2. For women of childbearing potential (WOCBP, see Annex VII for definition), non-agreement to follow instructions for method(s) of contraception for the heterosexual couple (see Annex VII for instructions) during the treatment period and follow-up, or at least 3 months after the last dose of IMP, or if there are concerns that they will not reliably comply with the contraception requirements.
3. Acute infection
4. aspartate aminotransferase (AST), or alanine aminotransferase (ALT), or total bilirubin \>1.5x (or \>2.5x in patients known for Gilbert's syndrome) the upper limit of normal
5. Renal clearance by Cockcroft-Gault formula \<30 ml/min
6. Untreated pulmonary arteriovenous malformation (if vaso-occlusion is technically feasible)
7. Hemoptysis or hematuria within the last 12 months
8. Ulcus or active gastric bleeding within the last 12 months
9. Anticoagulant or antiplatelets treatment
10. Coronary heart disease
11. Thrombotic event within the last 12 months
12. Long QT syndrome (on ECG performed at screening)
13. Known allergy to nintedanib, soya, peanuts
14. Bevacizumab, pazopanib or other anti-angiogenic treatments within the last 12 months
15. Concomitant treatment with ketoconazole, erythromycin, rifampicin, carbamazepine, phenytoin, St John's Wort
16. Surgery within the last 3 months or planned within the next 9 months
17. Recent unhealed wound
18. Any other serious underlying medical condition that could interfere with the study treatment and potential adverse events
19. Any mental or other impairment that may compromise compliance with the study requirements.
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