MedTrace Logo

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

NCT03954782 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 61

Last updated 2025-08-01

No results posted yet for this study

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

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)

Sponsors & Collaborators

  • Hospices Civils de Lyon

    lead OTHER

Principal Investigators

  • Sophie DUPUIS-GIROD · Hospices Civils de Lyon

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-06-22
Primary Completion
2023-02-24
Completion
2023-02-24

Countries

  • France

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03954782 on ClinicalTrials.gov