Using nintedanib to treat nosebleeds in patients with hereditary hemorrhagic telangiectasia
Phase II Multicentric Randomized Study on Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients
This study is testing if the medication nintedanib can help reduce the number and severity of nosebleeds in people with hereditary hemorrhagic telangiectasia.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 48 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Centre Hospitalier Universitaire Vaudois Academic / other |
| Drugs / interventions | Bevacizumab |
| Locations | 3 sites (Bron and 2 other locations) |
| Trial ID | NCT04976036 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of hereditary hemorrhagic telangiectasia and moderate to severe epistaxis.
Not a fit: Patients who are pregnant, breastfeeding, or have acute infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the frequency and severity of nosebleeds in patients with HHT, improving their quality of life.
How similar studies have performed: While nintedanib has been successful in treating other conditions, this specific application for epistaxis in HHT is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Bron and 2 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.