Hereditary Hemorrhagic TelangiectasiaPhase 3EPERONCT04113187
Propranolol for Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients
A Phase 3 study testing Propranolol treatment in people with Hereditary Hemorrhagic Telangiectasia and Osler Weber Rendu Disease, run by University Hospital, Bordeaux at 1 site in France.
Trial summary
Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder of angiogenesis associated with disabling epistaxis. Management of these nose bleedings requires more effective treatment. Propranolol, a beta-blocker, is a potentially useful therapeutic considering its anti-angiogenic properties. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia, Osler Weber Rendu Disease
- What is being tested?
- Propranolol treatment
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteFrance
- Study dates
- 23 Jun 2020to 19 May 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years
- Confirmed diagnosis of HHT : 3 or more Curaçao criteria (spontaneous and recurrent epistaxis; multiple telangiectasia at characteristic sites; visceral lesions such as gastrointestinal telangiectasia or arteriovenous malformations; family history: a first degree relative with HHT according to these criteria ) or mutations of genes encoding for ALK1, ENG or SMAD4
- Patient suffering from recurrent epistaxis (more than a mean of 10 episodes/month) and/or with a cumulative mean duration per month more than 20 minutes, according to specific grids completed at least three months before inclusion.
- Patient insured under the French social security system
- Free and informed consent signed by investigator and patient
Who cannot join
- Pregnancy or breast-feeding
- Incomplete epistaxis grids in the month prior inclusion
- Current beta-blocker treatment
- Hypersensitivity to the active substance or excipient
- Patients with type I or type II diabetes, treated with insulin, sulphonylureas or meglitinides
- Patients with heart failure
Show all 12Show fewer
- Patients with liver failure
- Patients with hepatic arteriovenous malformations responsible for high-output cardiac failure or severe hepatic dysfunction
- Patients with severe psoriasis (PASI>10)
- Contra-indication to beta-blocker treatment : asthma, chronic obstructive bronchopneumopathy, atrioventricular block of second or third degrees without pacemaker, Prinzmetal's angina, bradycardia < 50bpm, Raynaud's phenomenon, oblitering arteriopathy of the lower limbs, low blood pressure, non-treated pheochromocytoma
- Participation in another clinical therapeutic trial less than 3 months before inclusion
- Protected adult according to french law
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study of the Efficacy of Propranolol for the Management of Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients
Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder of angiogenesis associated with disabling epistaxis. Management of these nose bleedings requires more effective treatment. Propranolol, a beta-blocker, is a potentially useful therapeutic considering its anti-angiogenic properties. Our objective is to explore the efficacy of propranolol, three months after its introduction, on the cumulative duration of epistaxis in HHT patients.
Read the detailed description
Hereditary Hemorrhagic Telangiectasia (HHT) is a rare systemic autosomal dominantly inherited disorder of angiogenesis. Its major feature is the occurrence in 90% of patients of spontaneous and recurrent epistaxis responsible for iron deficiency and chronic anemia. Various conservative and interventional treatments have been described for these conditions, but no optimal therapy exists. Inhibiting angiogenesis process is an interesting therapeutic option. Propranolol, a non-cardio-selective beta-blocker, could represent a new candidate for the therapy of HHT telangiectasia as it suppresses angiogenesis in vitro. This anti-angiogenic property is well-known in pediatric dermatology, since C. Léauté-Labrèze and al. have demonstrated a great improvement of infantile hemangioma undergoing propranolol treatment. At the University Hospital Center of Bordeaux, the investigators assessed in a preliminary study the efficacy of propranolol for HHT epistaxis. Nine of ten patients receiving propranolol for cardiologic or neurologic indications, retrospectively analyzed, significantly improved their Epistaxis Severity Score. Ten patients were then prospectively included and after 3 months of propranolol treatment, the median duration of epistaxis per month significantly decreased (p=0,007) as well as the number of epistaxis episodes per month (p=0,015).
To confirm these results, the investigators would like to study the efficacy of propranolol given per os at the dose of 40 mg twice a day for a three-months period, in comparison to a placebo. Throughout the study, patients will complete specific grids recording the number of epistaxis episodes per month and the cumulative duration of nose bleedings. A follow-up of 6 months will be done (4 visits after inclusion), recording clinical and biological data and monitoring the tolerance of treatment.
- Being tested
- Propranolol treatment, Placebo
- Keywords
- angiogenesis, epistaxis, propranolol
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Epistaxis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Propranolol treatmentDrug
40 mg twice a day (morning and evening), per os, during three months
PlaceboDrug
per os, twice a day (morning and evening) during three months
| Study group | Role | What they receive |
|---|---|---|
| Propranolol arm | Experimental | Not described Receives: Drug: Propranolol treatment |
| Placebo arm | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative duration of epistaxis (in minutes)
Measured: 6 months after baseline (Day 0)
Secondary outcomes (9)
Frequency of epistaxis (number of episodes) per month
Measured: At baseline (Day 0), 3 months and 6 months after baseline
Number of cutaneous telangiectasia on hands and face
Measured: At baseline (Day 0), 3 months and 6 months after baseline.
Levels of hemoglobin
Measured: At baseline (Day 0), 3 months and 6 months after baseline.
Levels of ferritin
Measured: At baseline (Day 0), 3 months and 6 months after baseline.
Number of red blood cells transfusions
Measured: At baseline (Day 0), 3 months and 6 months after baseline.
Short Form (SF) 36 Health Survey
Measured: At baseline (Day 0), 3 months and 6 months after baseline.
Number of adverse events
Measured: 3 months and 6 months after baseline (Day 0).
Measurement of blood pressure
Measured: At baseline (Day 0), 1 month, 3 months and 6 months after baseline.
Measurement of heart rate
Measured: At baseline (Day 0), 1 month, 3 months and 6 months after baseline.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2019
- Study start23 Jun 2020
- Primary completion19 May 2022
- Study completion19 May 2022
Why it stopped: target number of subject not reached
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Hemorrhagic TelangiectasiaStudies of the same condition, open and under way.
- More trials run by University Hospital, BordeauxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bordeaux
- Sponsor type
- University, hospital or other
- Collaborators
- AMRO-HHT-France - Association Maladie de Rendu-Osler
- Responsible party
- Sponsor
- Investigators
- Anne CONTIS, MD, Principal investigator, University Hospital, BordeauxAntoine BENARD, MD, PhD, Study chair, University Hospital, Bordeaux
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04113187
- Sponsor's protocol ID
- CHUBX 2015/32
- Other IDs
- 2016-002770-12, 2016
- Acronym
- EPERO
- Status last verified
- Jun 2022
- First submitted
- 27 Sep 2019
- Last updated
- 23 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of the Efficacy of Propranolol for the Management of Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients
- ClinicalTrials.gov ID
- NCT04113187
- Acronym / sponsor ID
- EPERO / CHUBX 2015/32
- Keywords
- angiogenesis, epistaxis, propranolol
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: target number of subject not reached
- Enrolment
- 15 participants (actual)
- Lead sponsor
- University Hospital, BordeauxUniversity, hospital or other
- Collaborators
- AMRO-HHT-France - Association Maladie de Rendu-Osler
- Study start
- 23 Jun 2020 (actual)
- Primary completion
- 19 May 2022 (actual)
- Study completion
- 19 May 2022 (actual)
- First posted
- 2 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder of angiogenesis associated with disabling epistaxis. Management of these nose bleedings requires more effective treatment. Propranolol, a beta-blocker, is a potentially useful therapeutic considering its anti-angiogenic properties. Our objective is to explore the efficacy of propranolol, three months after its introduction, on the cumulative duration of epistaxis in HHT patients.
Detailed description
Hereditary Hemorrhagic Telangiectasia (HHT) is a rare systemic autosomal dominantly inherited disorder of angiogenesis. Its major feature is the occurrence in 90% of patients of spontaneous and recurrent epistaxis responsible for iron deficiency and chronic anemia. Various conservative and interventional treatments have been described for these conditions, but no optimal therapy exists. Inhibiting angiogenesis process is an interesting therapeutic option. Propranolol, a non-cardio-selective beta-blocker, could represent a new candidate for the therapy of HHT telangiectasia as it suppresses angiogenesis in vitro. This anti-angiogenic property is well-known in pediatric dermatology, since C. Léauté-Labrèze and al. have demonstrated a great improvement of infantile hemangioma undergoing propranolol treatment. At the University Hospital Center of Bordeaux, the investigators assessed in a preliminary study the efficacy of propranolol for HHT epistaxis. Nine of ten patients receiving propranolol for cardiologic or neurologic indications, retrospectively analyzed, significantly improved their Epistaxis Severity Score. Ten patients were then prospectively included and after 3 months of propranolol treatment, the median duration of epistaxis per month significantly decreased (p=0,007) as well as the number of epistaxis episodes per month (p=0,015).
To confirm these results, the investigators would like to study the efficacy of propranolol given per os at the dose of 40 mg twice a day for a three-months period, in comparison to a placebo. Throughout the study, patients will complete specific grids recording the number of epistaxis episodes per month and the cumulative duration of nose bleedings. A follow-up of 6 months will be done (4 visits after inclusion), recording clinical and biological data and monitoring the tolerance of treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Propranolol arm | Experimental | Drug: Propranolol treatment |
| Placebo arm | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18 years
- Confirmed diagnosis of HHT : 3 or more Curaçao criteria (spontaneous and recurrent epistaxis; multiple telangiectasia at characteristic sites; visceral lesions such as gastrointestinal telangiectasia or arteriovenous malformations; family history: a first degree relative with HHT according to these criteria ) or mutations of genes encoding for ALK1, ENG or SMAD4
- Patient suffering from recurrent epistaxis (more than a mean of 10 episodes/month) and/or with a cumulative mean duration per month more than 20 minutes, according to specific grids completed at least three months before inclusion.
- Patient insured under the French social security system
- Free and informed consent signed by investigator and patient
Key exclusion criteria (12 listed)
- Pregnancy or breast-feeding
- Incomplete epistaxis grids in the month prior inclusion
- Current beta-blocker treatment
- Hypersensitivity to the active substance or excipient
- Patients with type I or type II diabetes, treated with insulin, sulphonylureas or meglitinides
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Propranolol treatment40 mg twice a day (morning and evening), per os, during three months | Drug | Propranolol arm |
| Placeboper os, twice a day (morning and evening) during three months | Drug | Placebo arm |
Primary outcome
Cumulative duration of epistaxis (in minutes)
Time frame: 6 months after baseline (Day 0)
9 secondary outcomes
Frequency of epistaxis (number of episodes) per month
Time frame: At baseline (Day 0), 3 months and 6 months after baseline
Number of cutaneous telangiectasia on hands and face
Time frame: At baseline (Day 0), 3 months and 6 months after baseline.
Levels of hemoglobin
Time frame: At baseline (Day 0), 3 months and 6 months after baseline.
Levels of ferritin
Time frame: At baseline (Day 0), 3 months and 6 months after baseline.
Number of red blood cells transfusions
Time frame: At baseline (Day 0), 3 months and 6 months after baseline.
Short Form (SF) 36 Health Survey
Time frame: At baseline (Day 0), 3 months and 6 months after baseline.
Number of adverse events
Time frame: 3 months and 6 months after baseline (Day 0).
Measurement of blood pressure
Time frame: At baseline (Day 0), 1 month, 3 months and 6 months after baseline.
Measurement of heart rate
Time frame: At baseline (Day 0), 1 month, 3 months and 6 months after baseline.
Results and safety
The study was stopped early (reason given: target number of subject not reached). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anne CONTIS, MD principal investigator
University Hospital, Bordeaux
Antoine BENARD, MD, PhD study chair
University Hospital, Bordeaux
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Propranolol treatment in people with Hereditary Hemorrhagic Telangiectasia and Osler Weber Rendu Disease, run by University Hospital, Bordeaux at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: target number of subject not reached). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative duration of epistaxis (in minutes) (time frame: 6 months after baseline (Day 0)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2026.