Hereditary Hemorrhagic Telangiectasia (HHT)CoBéNCT07772050
IV Bevacizumab Treatment of Patients With Hereditary Hemorrhagic Telangiectasia (HHT) or Rendu-Osler (RO) Disease: A Retrospective Analysis of French Cohort Data From 2009 to 2024
An observational study of Hereditary Hemorrhagic Telangiectasia (HHT) and Rendu Osler Disease, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Rendu-Osler disease (RO), also known as Hereditary Haemorrhagic Telangiectasia (HHT), is an autosomal dominant genetic disorder. Its pathophysiology is associated with an imbalance in angiogenic balance. The resulting epistaxis and digestive bleeding can be severe, requiring repeated transfusions in 5% of patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia (HHT), Rendu Osler Disease
- What is being tested?
- Real World data study.
- Type of treatment
- Injection or infusion
- How many people
- 244 participants
- Where
- 1 siteFrance
- Study dates
- 1 Jan 2009to 31 Dec 2025
- Phase
- Observational
- 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
Study population: A Total of 317 patients will be included , all of whom were adults aged 18 years or older, diagnosed with hereditary haemorrhagic telangiectasia, and have received systemic bevacizumab exclusively for HHT-related manifestations.
Who may be able to join
- - Adult over 18 years of age
- Rendu-Osler disease treated with bevacizumab prescribed for this condition
- Treated and followed in France
Who cannot join
- - Patient opposition to data use
Trial locations and contacts
1 site in 1 country.
About this study
Official title: IV Bevacizumab Treatment of Patients With Hereditary Hemorrhagic Telangiectasia or Rendu-Osler Disease: A Retrospective Analysis of French Cohort Data From 2009 to 2024
Rendu-Osler disease (RO), also known as Hereditary Haemorrhagic Telangiectasia (HHT), is an autosomal dominant genetic disorder. Its pathophysiology is associated with an imbalance in angiogenic balance. The resulting epistaxis and digestive bleeding can be severe, requiring repeated transfusions in 5% of patients. In addition, some patients with HHT may present with liver damage, leading to high-flow heart failure.
Vascular Endothelial Growth Factor (VEGF) plays a role in the pathophysiology of Rendu-Osler disease, leading to the use of a recombinant anti-VEGF monoclonal antibody, bevacizumab. Studies conducted and promoted by HCL have demonstrated the efficacy and safety of bevacizumab in patients with HHT.
Read more
In 2014, bevacizumab was granted orphan drug designation for this disease. The 2020 international guidelines for the diagnosis and management of HHT suggest the use of systemic antiangiogenic agents in cases of epistaxis resistant to other treatments. The use of bevacizumab has been widely published in uncontrolled studies, demonstrating its efficacy in reducing epistaxis, improving anaemia, reducing transfusion dependence and improving quality of life.
In France, bevacizumab is currently prescribed outside the scope of its marketing authorization. Treatment indications are validated within the French network of expert centres and the FAVAmulti rare disease network. In other countries, access to this treatment varies widely according to local regulations, and is currently being evaluated in the European VASCERN network.
The aim of this study is to describe the use of bevacizumab in patients treated for HHT in France.
- Being tested
- Real World data study.
- Keywords
- HHT, Rendu-Osler, Bevacizumab, Bleeding, High Cardiac output, Anaemia
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Hemorrhage, Cardiac Output, High, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Real World data study.Other
The bevacizumab population is defined as patients who received at least one bevacizumab injection. Descriptive analyses will be performed using demographic, clinical and genetic data
| Study group | Role | What they receive |
|---|---|---|
| HHT patients treated with IV bevacizumab | Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe HHT-related bleeding or severe hepatic involvement, consistent with established recommendations Receives: Other: Real World data study. | |
| HHT patients treated with IV bevacizumab for severe bleeding | Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe HHT-related bleeding consistent with established recommendations Receives: Other: Real World data study. | |
| HHT patients treated with IV bevacizumab for hight cardiac output/cardiac failure secondary to sever | Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe hepatic involvement, consistent with established recommendations Receives: Other: Real World data study. | |
| HHT patients treated with IV bevacizumab for severe bleeding (epistaxis, Gastrointestinal bleeding G | Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe HHT-related bleeding and severe hepatic involvement, consistent with established recommendations Receives: Other: Real World data study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The cumulated number of RBC (Red Blood Cell) units transfused
The number of RBC units transfused (date and number of units)
Measured: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel
Secondary outcomes (1)
Hemoglobin values (g/L)
Hemoglobin values were collected continually, the average of all available hemoglobin values were calculated for each period, anemia was defined as an hemoglobin mean level less than 120 g/dl regardless of gender
Measured: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2026
- Study start1 Jan 2009
- Primary completion31 Dec 2024
- Study completion31 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Hemorrhagic Telangiectasia (HHT)Studies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 244 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07772050
- Sponsor's protocol ID
- 24-5247
- Acronym
- CoBé
- Status last verified
- May 2026
- First submitted
- 19 Mar 2026
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- IV Bevacizumab Treatment of Patients With Hereditary Hemorrhagic Telangiectasia or Rendu-Osler Disease: A Retrospective Analysis of French Cohort Data From 2009 to 2024
- ClinicalTrials.gov ID
- NCT07772050
- Acronym / sponsor ID
- CoBé / 24-5247
- Keywords
- HHT, Rendu-Osler, Bevacizumab, Bleeding, High Cardiac output, Anaemia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 244 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 1 Jan 2009 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 31 Dec 2025 (actual)
- First posted
- 19 Aug 2026 (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.
Rendu-Osler disease (RO), also known as Hereditary Haemorrhagic Telangiectasia (HHT), is an autosomal dominant genetic disorder. Its pathophysiology is associated with an imbalance in angiogenic balance. The resulting epistaxis and digestive bleeding can be severe, requiring repeated transfusions in 5% of patients. In addition, some patients with HHT may present with liver damage, leading to high-flow heart failure.
Vascular Endothelial Growth Factor (VEGF) plays a role in the pathophysiology of Rendu-Osler disease, leading to the use of a recombinant anti-VEGF monoclonal antibody, bevacizumab. Studies conducted and promoted by HCL have demonstrated the efficacy and safety of bevacizumab in patients with HHT.
In 2014, bevacizumab was granted orphan drug designation for this disease. The 2020 international guidelines for the diagnosis and management of HHT suggest the use of systemic antiangiogenic agents in cases of epistaxis resistant to other treatments. The use of bevacizumab has been widely published in uncontrolled studies, demonstrating its efficacy in reducing epistaxis, improving anaemia, reducing transfusion dependence and improving quality of life.
In France, bevacizumab is currently prescribed outside the scope of its marketing authorization. Treatment indications are validated within the French network of expert centres and the FAVAmulti rare disease network. In other countries, access to this treatment varies widely according to local regulations, and is currently being evaluated in the European VASCERN network.
The aim of this study is to describe the use of bevacizumab in patients treated for HHT in France.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| HHT patients treated with IV bevacizumab | – | Other: Real World data study.Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe HHT-related bleeding or severe hepatic involvement, consistent with established recommendations |
| HHT patients treated with IV bevacizumab for severe bleeding | – | Other: Real World data study.Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe HHT-related bleeding consistent with established recommendations |
| HHT patients treated with IV bevacizumab for hight cardiac output/cardiac failure secondary to sever | – | Other: Real World data study.Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe hepatic involvement, consistent with established recommendations |
| HHT patients treated with IV bevacizumab for severe bleeding (epistaxis, Gastrointestinal bleeding G | – | Other: Real World data study.Patients from the CIROCO database: Adults aged 18 years and older, diagnosed with HHT and have received bevacizumab between 2009 and 2025, in France, for severe HHT-related bleeding and severe hepatic involvement, consistent with established recommendations |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- A Total of 317 patients will be included , all of whom were adults aged 18 years or older, diagnosed with hereditary haemorrhagic telangiectasia, and have received systemic bevacizumab exclusively for HHT-related manifestations.
Key inclusion criteria (3 listed)
- - Adult over 18 years of age
- Rendu-Osler disease treated with bevacizumab prescribed for this condition
- Treated and followed in France
Key exclusion criteria (1 listed)
- - Patient opposition to data use
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 |
|---|---|---|
| Real World data study.The bevacizumab population is defined as patients who received at least one bevacizumab injection. Descriptive analyses will be performed using demographic, clinical and genetic data | Other | HHT patients treated with IV bevacizumab, HHT patients treated with IV bevacizumab for hight cardiac output/cardiac failure secondary to sever, HHT patients treated with IV bevacizumab for severe bleeding, HHT patients treated with IV bevacizumab for severe bleeding (epistaxis, Gastrointestinal bleeding G |
Primary outcome
The cumulated number of RBC (Red Blood Cell) units transfused
Time frame: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel
The number of RBC units transfused (date and number of units)
1 secondary outcome
Hemoglobin values (g/L)
Time frame: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2026.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Hemorrhagic Telangiectasia (HHT) and Rendu Osler Disease, run by Hospices Civils de Lyon 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 3 inclusion and 1 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: finished, as of the sponsor's last update on 19 Aug 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The cumulated number of RBC (Red Blood Cell) units transfused (time frame: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel). 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 19 Aug 2026.