Using nintedanib to treat nosebleeds in patients with hereditary hemorrhagic telangiectasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the efficacy of nintedanib, an antifibrotic medication, in reducing the frequency and severity of epistaxis (nosebleeds) in patients with hereditary hemorrhagic telangiectasia (HHT). The study will involve approximately 48 participants who will undergo an initial 2-month observation period followed by a 16-week treatment phase where they will receive either nintedanib or a placebo. Participants will keep a diary to record their epistaxis episodes throughout the study duration of 8 months. The trial aims to provide insights into a potential new treatment for a condition that currently lacks effective management options.

Why it matters

Who can join: the full rules

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.

Where it's running

• Lyon University Hospital, Dpt of genetics
  Bron, France Recruiting
• Clermont-Ferrand university hospital
  Clermont-Ferrand, France Recruiting
• Angiology Department, Lausanne University Hospital
  Lausanne, Canton of Vaud, Switzerland Completed

Study contacts

• Study coordinator: Romain Lazor, MD
• Email: romain.lazor@chuv.ch
• Phone: +41 79 556 21 69

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Registry for patients with Hereditary Hemorrhagic Telangiectasia

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.