Hereditary Hemorrhagic TelangiectasiaNCT04150822
CHORUS - Comprehensive HHT Outcomes Registry of the United States (Formerly OUR HHT Registry)
An observational study of Hereditary Hemorrhagic Telangiectasia and HHT, run by Unity Health Toronto at 1 site in Canada.
Trial summary
The goal of this study is to better understand HHT, the symptoms and complications it causes ("outcomes") and how the disease impacts people's lives. The investigators are aiming to recruit and gather information together in the Registry from 1,000 HHT patients from four HHT Centres of Excellence in North America. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia, HHT, Arteriovenous Malformation of Brain
- What is being tested?
- Registry and Saliva sample
- How many people
- 296 participants
- Where
- 1 siteCanada
- Study dates
- 1 Nov 2018to 30 Jun 2028
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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: Subjects with Hereditary Hemorrhagic Telangiectasia (HHT), living in North America
Who may be able to join
- Participants diagnosed with HHT by the Curacao criteria (either 3+ clinical diagnostic criteria or genetic diagnosis).
- Capable of giving informed consent in person or via a substitute decision maker
- >18 years
Who cannot join
- Participants unable to give informed consent either in person or with a substitute decision maker
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Hereditary Hemorrhagic Telangiectasia (HHT) Research Outcomes Registry
The goal of this study is to better understand HHT, the symptoms and complications it causes ("outcomes") and how the disease impacts people's lives. The investigators are aiming to recruit and gather information together in the Registry from 1,000 HHT patients from four HHT Centres of Excellence in North America. The Investigators will collect long-term information about the people in the Registry, allowing the investigators to understand how the disease changes over time, and what factors can influence those changes. Ultimately, this should help improve treatments for the disease.
Read the detailed description
Background and Rationale HHT has an estimated prevalence of 1 in 5000, affecting children and adults, in multiple organs. The disease is characterized by the presence of vascular malformations (VMs), including arteriovenous malformations (AVMs) of the lung, liver, brain, spinal cord and smaller mucosal lesions (telangiectasia) of the nose, mouth and GI tract. These lesions lead to acute and chronic bleeding, stroke, heart failure and death. Treatments are currently mostly limited to managing complications, while approximately 90% of adults have ongoing symptoms, despite best surgical and medical therapies. With recent drug development related to angiogenesis, there is hope for effective novel therapies. Investigators, experts, the International HHT Guidelines, Pharma representatives, the CDC and HHT patient advocates (curehht.org) all agree that there is an urgent need for natural history data in this disease, with characterization of clinical outcomes, to allow patients to benefit from the explosion of drug development in the field.
As of August 27, 2023, the OUR HHT Registry has transitioned to the CHORUS platform (Studytrax) for ongoing data collection. Only select CHORUS-funded sites are currently enrolling. The Toronto site has completed enrollment but remains active with REB approval.
- Being tested
- Registry and Saliva sample
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Arteriovenous Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Registry and Saliva sampleOther
Non-interventional registry with saliva sample collected for DNA analysis
What is being measured
The primary outcome is the main question the study is designed to answer.
Prospective and longitudinal characterization of major outcomes of HHT in a cohort of HHT patients, from Centers of Excellence in North America.
Comprehensive baseline clinical, demographic and lifestyle data will be collected and entered into the recruitment-ready newly developed OUR HHT Registry.
Measured: 10 Years
The longitudinal characterization of major outcomes of HHT in the North American cohort
Annual outcome data will be collected and entered into the recruitment-ready newly developed OUR HHT Registry.
Measured: 10 Years
Secondary outcomes (5)
Characterizing the determinants of HHT by prospectively and longitudinally measuring the rates of clinical outcome of HHT
The rates of severe complications of HHT will be measured and their determinants characterized.
Measured: 10 Years
Epistaxis which affects 90% of adults with HHT, will be characterized by measuring the rates of clinical outcome.
The characteristics and determinants of epistaxis will be studied.
Measured: 10 Years
The prospective development of organ VMs in HHT patients
Development of new VMs/growth of VMs and its determinants will be measured.
Measured: 10 Years
The rates of venous thromboembolism (VTE) in HHT patients
The rates and determinants of venous thromboembolism in HHT patients will be measured prospectively.
Measured: 10 Years
A DNA repository of HHT subjects will be created as a resource for future genetic, pharmacogenetics and targeted therapy studies.
Saliva samples of all recruited subjects will collected to create a DNA repository
Measured: 10 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2019
- Study start1 Nov 2018
- Primary completion23 Jun 2023
- Study completion30 Jun 2028 (estimated)
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 Unity Health TorontoThe 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
- Prospective
- Enrolment
- 296 participants (actual)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Unity Health Toronto
- Sponsor type
- University, hospital or other
- Collaborators
- Cure HHT, Dartmouth College
- Responsible party
- Sponsor
- Investigators
- Marie E Faughnan, MD, Principal investigator, Unity Health Toronto
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04150822
- Sponsor's protocol ID
- OURHHT
- Status last verified
- Apr 2026
- First submitted
- 18 Sep 2019
- Last updated
- 22 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 22 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hereditary Hemorrhagic Telangiectasia (HHT) Research Outcomes Registry
- ClinicalTrials.gov ID
- NCT04150822
- Acronym / sponsor ID
- OURHHT
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 296 participants (actual)
- Lead sponsor
- Unity Health TorontoUniversity, hospital or other
- Collaborators
- Cure HHT, Dartmouth College
- Study start
- 1 Nov 2018 (actual)
- Primary completion
- 23 Jun 2023 (actual)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 5 Nov 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.
The goal of this study is to better understand HHT, the symptoms and complications it causes ("outcomes") and how the disease impacts people's lives. The investigators are aiming to recruit and gather information together in the Registry from 1,000 HHT patients from four HHT Centres of Excellence in North America. The Investigators will collect long-term information about the people in the Registry, allowing the investigators to understand how the disease changes over time, and what factors can influence those changes. Ultimately, this should help improve treatments for the disease.
Detailed description
Background and Rationale HHT has an estimated prevalence of 1 in 5000, affecting children and adults, in multiple organs. The disease is characterized by the presence of vascular malformations (VMs), including arteriovenous malformations (AVMs) of the lung, liver, brain, spinal cord and smaller mucosal lesions (telangiectasia) of the nose, mouth and GI tract. These lesions lead to acute and chronic bleeding, stroke, heart failure and death. Treatments are currently mostly limited to managing complications, while approximately 90% of adults have ongoing symptoms, despite best surgical and medical therapies. With recent drug development related to angiogenesis, there is hope for effective novel therapies. Investigators, experts, the International HHT Guidelines, Pharma representatives, the CDC and HHT patient advocates (curehht.org) all agree that there is an urgent need for natural history data in this disease, with characterization of clinical outcomes, to allow patients to benefit from the explosion of drug development in the field.
As of August 27, 2023, the OUR HHT Registry has transitioned to the CHORUS platform (Studytrax) for ongoing data collection. Only select CHORUS-funded sites are currently enrolling. The Toronto site has completed enrollment but remains active with REB approval.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Subjects with Hereditary Hemorrhagic Telangiectasia (HHT), living in North America
Key inclusion criteria (3 listed)
- Participants diagnosed with HHT by the Curacao criteria (either 3+ clinical diagnostic criteria or genetic diagnosis).
- Capable of giving informed consent in person or via a substitute decision maker
- >18 years
Key exclusion criteria (1 listed)
- Participants unable to give informed consent either in person or with a substitute decision maker
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 |
|---|---|---|
| Registry and Saliva sampleNon-interventional registry with saliva sample collected for DNA analysis | Other | – |
Primary outcome
Prospective and longitudinal characterization of major outcomes of HHT in a cohort of HHT patients, from Centers of Excellence in North America.
Time frame: 10 Years
Comprehensive baseline clinical, demographic and lifestyle data will be collected and entered into the recruitment-ready newly developed OUR HHT Registry.
The longitudinal characterization of major outcomes of HHT in the North American cohort
Time frame: 10 Years
Annual outcome data will be collected and entered into the recruitment-ready newly developed OUR HHT Registry.
5 secondary outcomes
Characterizing the determinants of HHT by prospectively and longitudinally measuring the rates of clinical outcome of HHT
Time frame: 10 Years
Epistaxis which affects 90% of adults with HHT, will be characterized by measuring the rates of clinical outcome.
Time frame: 10 Years
The prospective development of organ VMs in HHT patients
Time frame: 10 Years
The rates of venous thromboembolism (VTE) in HHT patients
Time frame: 10 Years
A DNA repository of HHT subjects will be created as a resource for future genetic, pharmacogenetics and targeted therapy studies.
Time frame: 10 Years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 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.
Marie E Faughnan, MD principal investigator
Unity Health Toronto
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 and HHT, run by Unity Health Toronto at 1 site in Canada.
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: under way, not looking for participants, as of the sponsor's last update on 22 Apr 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 Canada. 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?
This study is still under way. Results are not available in the source record yet.
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?
Prospective and longitudinal characterization of major outcomes of HHT in a cohort of HHT patients, from Centers of Excellence in North America. (time frame: 10 Years). The study lists 2 primary outcomes in all. 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 22 Apr 2026.