Phase II Monocentric Randomized Study on Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients

Who is this study for? Patients with Hereditary Hemorrhagic Telangiectasia
What treatments are being studied? Nintedanib
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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 the study, i.e. 8 months. After 2 months of observation, the treatment phase will begin. Participants will take a capsule (nintedanib 150 mg or placebo) once a day for 2 weeks, then twice a day for 14 weeks. In case of intolerance at the dose of 2 capsules per day, the treatment may be reduced to 1 capsule per day. Subjects will also have to mention on the diary any blood transfusion, iron perfusion, and any symptoms they may be experiencing. Following the 16 weeks of treatment, an 8-week follow-up period will allow to observe the effects of nintedanib after the end of the treatment period, and to monitor any unexpected adverse events.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• signed informed consent

• 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)

• age ≥18 years at the time of informed consent

• moderate to serious epistaxis defined as Epistaxis Severity Score (ESS) ≥2.5

• absence of cerebral arteriovenous malformation demonstrated by brain imaging

Locations
Other Locations
Switzerland
Respiratory medicine Department, Lausanne University Hospital
RECRUITING
Lausanne
Contact Information
Primary
Romain Lazor, MD
romain.lazor@chuv.ch
+41213144746
Backup
Estelle Clement, RN
estelle.clement@chuv.ch
Time Frame
Start Date: 2022-05-05
Estimated Completion Date: 2026-07
Participants
Target number of participants: 48
Treatments
Experimental: Nintedanib
nintedanib 150 mg once a day for 2 weeks, then twice a day for 14 weeks
Placebo_comparator: Placebo
placebo 150 mg once a day for 2 weeks, then twice a day for 14 weeks
Sponsors
Leads: Dr. Romain Lazor
Collaborators: Boehringer Ingelheim

This content was sourced from clinicaltrials.gov