- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04150822
Hereditary Hemorrhagic Telangiectasia (HHT) Research Outcomes Registry (HHT)
March 4, 2024 updated by: Unity Health Toronto
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.
Study Overview
Status
Active, not recruiting
Intervention / Treatment
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.
Study Type
Observational
Enrollment (Actual)
287
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Dewi S Clark, BSc, MHSc
- Phone Number: 42887 416-360-4000
- Email: dewi.clark@unityhealth.to
Study Locations
-
-
Ontario
-
Toronto, Ontario, Canada, M5B1W8
- St. Michael's Hospital
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Sampling Method
Probability Sample
Study Population
Subjects with Hereditary Hemorrhagic Telangiectasia (HHT), living in North America
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- Participants unable to give informed consent either in person or with a substitute decision maker
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
10 Years
|
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.
|
10 Years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Characterizing the determinants of HHT by prospectively and longitudinally measuring the rates of clinical outcome of HHT
Time Frame: 10 Years
|
The rates of severe complications of HHT will be measured and their determinants characterized.
|
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 characteristics and determinants of epistaxis will be studied.
|
10 Years
|
The prospective development of organ VMs in HHT patients
Time Frame: 10 Years
|
Development of new VMs/growth of VMs and its determinants will be measured.
|
10 Years
|
The rates of venous thromboembolism (VTE) in HHT patients
Time Frame: 10 Years
|
The rates and determinants of venous thromboembolism in HHT patients will be measured prospectively.
|
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
|
Saliva samples of all recruited subjects will collected to create a DNA repository
|
10 Years
|
Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Participant entered data
Time Frame: 10 Years
|
Data collection from participant relating symptoms and knowledge of HHT
|
10 Years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Marie E Faughnan, MD, Unity Health Toronto
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 1, 2018
Primary Completion (Estimated)
December 1, 2028
Study Completion (Estimated)
December 1, 2028
Study Registration Dates
First Submitted
September 18, 2019
First Submitted That Met QC Criteria
October 31, 2019
First Posted (Actual)
November 5, 2019
Study Record Updates
Last Update Posted (Actual)
March 5, 2024
Last Update Submitted That Met QC Criteria
March 4, 2024
Last Verified
March 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Cerebrovascular Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Congenital Abnormalities
- Hematologic Diseases
- Hemorrhagic Disorders
- Hemostatic Disorders
- Cardiovascular Abnormalities
- Nervous System Malformations
- Vascular Malformations
- Intracranial Arterial Diseases
- Central Nervous System Vascular Malformations
- Arteriovenous Malformations
- Telangiectasis
- Telangiectasia, Hereditary Hemorrhagic
- Intracranial Arteriovenous Malformations
Other Study ID Numbers
- OURHHT
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
UNDECIDED
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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