NCT03954782

Efficacy of Nintedanib Per os as a Treatment for Epistaxis in HHT Disease.

Efficacy of Nintedanib Per os as a Treatment for Epistaxis in HHT Disease. A National, Randomized, Multicentre Phase II Study

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 61 (actual)
• Sponsor: Hospices Civils de Lyon · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The recognized manifestations of HHT are all due to abnormalities in vascular structure. Epistaxis are spontaneous, very variable, may occur as often as several times every day, and are recurrent in 90% of patients and associated with chronic and severe anemia in 2-10%. They also significantly reduce quality of life. Blood transfusions are sometimes required in 10-30% of patients. Previous studies showed that antiangiogenic treatments such as anti-VEGF treatment (bevacizumab) administered intravenously was efficient on epistaxis and dramatically reduced nosebleeds. Tyrosine kinase inhibitors are anti-angiogenic molecules which are available orally and could therefore overcome the difficulties encountered with bevacizumab. The investigator hypothesized that nintedanib, acting by indirect inhibition of the VEGF receptor should allow a reduction of epistaxis in HHT patient. Nintedanib has been used in one HHT patient following the diagnosis of Insterstitial Pulmonary Fibrosis (published case report in 2017, Kovacs et al) with encouraging results. The aim is to evaluate efficacy of nintedanib for the treatment of epistaxis in HHT patients

Conditions

• Telangiectasia, Hereditary Hemorrhagic
• Rendu Osler Disease

Interventions

Type	Name	Description
DRUG	Nintedanib 150 mg and 100 mg soft capsules	Nintedanib 150 mg soft capsules twice daily approximately 12 hours apart (i.e. 300 mg/day) for 12 weeks. In case of adverse reaction a dose reduction at 200 mg/day (100 mg twice daily) can be prescribe.
DRUG	Oral treatment of placebo soft capsule	Placebo soft capsules (identical to 150 mg and 100 mg soft capsules)

Timeline

• Start date: 2020-06-22
• Primary completion: 2023-02-24
• Completion: 2023-02-24
• First posted: 2019-05-17
• Last updated: 2025-08-01

Locations

8 sites across 1 country: France