Future treatments for hereditary hemorrhagic telangiectasia.
Florian Robert, Agnès Desroches-Castan, Sabine Bailly...
https://pubmed.ncbi.nlm.nih.gov/31910860Actively Recruiting
Phase 2
Age: 18Years +
All Genders
ID04976036
Phase II Randomized Study of Nintedanib for Treating Nosebleeds in Hereditary Hemorrhagic Telangiectasia Patients
Led by Dr. Romain Lazor · Updated on 2026-03-11
48
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
Sponsors
D
Dr. Romain Lazor
Lead Sponsor
B
Boehringer Ingelheim
Collaborating Sponsor
AI-Summary
What this Trial Is About
Hereditary hemorrhagic telangiectasia (HHT) is a rare genetic condition causing abnormal blood vessel formation, leading to frequent and severe nosebleeds called epistaxis. This trial evaluates the effect of the drug nintedanib, which targets blood vessel growth pathways, to see if it can reduce the frequency and duration of nosebleeds in HHT patients. The study is a phase II randomized controlled trial designed to test this hypothesis with about 48 participants. Participants will first undergo 2 months of observation where they record daily nosebleeds in a diary. Then, in the treatment phase lasting 16 weeks, they will take either nintedanib capsules or a placebo once daily for 2 weeks, then twice daily for 14 weeks, with dose reductions allowed if side effects occur. After treatment, an 8-week follow-up will monitor any lasting effects or adverse events. Both groups will have the same assessments throughout the study. During the study, participants will complete daily diaries noting nosebleed episodes, transfusions, iron infusions, and symptoms for the entire 8 months. They will have blood tests to measure hemoglobin, ferritin, and liver and kidney function at multiple time points. Quality of life and nosebleed severity scores will also be collected at various visits. Skin imaging of telangiectasia will be done at select visits. Safety will be closely monitored, and emergency contact is provided. Data confidentiality is ensured, and results will be securely stored.
CONDITIONS
Brief Title
Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Signed informed consent
- Definite diagnosis of hereditary hemorrhagic telangiectasia (HHT) by genetic mutation or clinical criteria
- Age 18 years or older at consent
- Moderate to serious nosebleeds with an Epistaxis Severity Score of 2.5 or higher
- No cerebral arteriovenous malformation shown by brain imaging
You will not qualify if you...
- Women who are pregnant or breastfeeding
- Women of childbearing potential not agreeing to contraception during and 3 months after treatment
- Acute infection
- Liver enzymes or bilirubin levels above specified limits
- Kidney clearance below 30 ml/min
- Untreated pulmonary arteriovenous malformations if treatable
- Hemoptysis or hematuria within last 12 months
- Active stomach ulcers or bleeding within last 12 months
- Use of anticoagulant or antiplatelet medications
- Coronary heart disease
- Thrombotic event within last 12 months
- Long QT syndrome on screening ECG
- Allergy to nintedanib, soya, or peanuts
- Use of certain anti-angiogenic drugs within last 12 months
- Use of specific interacting medications
- Surgery within last 3 months or planned within next 9 months
- Recent unhealed wounds
- Serious medical conditions interfering with treatment
- Mental or other impairments affecting study compliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Pre-therapeutic Observation Period
Duration - 8 weeks
Participants are observed without treatment to monitor their baseline epistaxis and health status.
Visits at screening, week 8, and periodic assessments including daily self-administered epistaxis assessment
Treatment
Duration - 16 weeks
Participants take either nintedanib or placebo according to the assigned regimen to evaluate the effect on epistaxis.
Visits at weeks 8, 12, and 16 with blood sampling and assessments; daily self-administered epistaxis assessment throughout
Follow-up
Duration - 8 weeks
Participants are monitored after treatment to assess post-treatment effects and possible adverse events.
Visits at weeks 20, 24, and 32 with blood sampling and assessments
Trial Site Locations
Total: 3 locations
1
Lyon University Hospital, Dpt of genetics
Bron, France, 69677
Actively Recruiting
2
Clermont-Ferrand university hospital
Clermont-Ferrand, France, 63000
Actively Recruiting
3
Angiology Department, Lausanne University Hospital
Lausanne, Canton of Vaud, Switzerland, 1011
Completed
Loading map...
Research Team
R
Romain Lazor, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Published Research Related To This Trial
Nintedanib as a novel treatment option in hereditary haemorrhagic telangiectasia.
Evelin Kovacs-Sipos, David Holzmann, Thomas Scherer...
https://pubmed.ncbi.nlm.nih.gov/28652319