NCT06259292

Brief Summary

The Comprehensive HHT Outcomes Registry of the United States (CHORUS) is an observational registry of patients diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT). The purpose of this study is to better understand HHT, the symptoms and complications it causes, and the impact the disease has on people's lives. The investigators will collect long-term information about the participant, allowing us to understand how the disease changes over time, and what factors can influence those changes. Ultimately, this should help improve treatments for the disease. Another important goal of the study is to provide a way to contact people to participate in future clinical trials and other research. The registry will be a centralized resource for recruitment for clinical trials. People in the registry will not be obligated to join any of these additional studies, but if interested, can agree to be contacted if they may be eligible for a study. Participants will:

  • Be asked to provide permission to collect information from their medical records, including things like demographic information, diagnosis information, family history, test results, treatment information, symptoms, complications, lifestyle and other relevant medical information.
  • Be asked study-related questions by phone or at a clinic visit.
  • Be asked study-related questions every year after enrollment for up to 10 years or until the study ends. A member of the study team will communicate with participants by phone or at clinic visits to collect information regarding any changes to their health over the previous year/s including new test results, treatment information, symptoms, and complications from HHT.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10,000

participants targeted

Target at P75+ for all trials

Timeline
91mo left

Started Nov 2023

Longer than P75 for all trials

Geographic Reach
1 country

16 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress25%
Nov 2023Nov 2033

Study Start

First participant enrolled

November 13, 2023

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

January 22, 2024

Completed
23 days until next milestone

First Posted

Study publicly available on registry

February 14, 2024

Completed
9.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2033

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2033

Last Updated

June 13, 2025

Status Verified

June 1, 2025

Enrollment Period

10 years

First QC Date

January 22, 2024

Last Update Submit

June 10, 2025

Conditions

Outcome Measures

Primary Outcomes (2)

  • Comprehensive Baseline Data Collection using HHT Baseline Assessment Scale

    Utilizing the "HHT Baseline Assessment Scale," this measure focuses on recruiting a diverse cohort of HHT patients across North America. Comprehensive baseline clinical, demographic, and lifestyle data will be collected and entered into CHORUS. The data, aggregated as mean change from baseline using the scale, will serve as a foundational resource for future clinical translational studies, addressing the urgent need for natural history data in HHT.

    10 years

  • Prospective Longitudinal Clinical Outcomes Assessment using HHT Clinical Outcomes Scale

    Utilizing the "HHT Clinical Outcomes Scale," this measure aims to prospectively and longitudinally assess the clinical outcomes of HHT patients. Determinants such as demographic factors, environmental influences, lifestyle choices, comorbidities, medications, HHT-genotype, and organ vascular malformations (VMs) will be measured. The data, aggregated as mean change from baseline using the scale, will enhance understanding and accelerate the development of new diagnostic and treatment options for HHT.

    10 years

Secondary Outcomes (4)

  • Identify severe morbidity and mortality outcomes related to HHT using HHT Clinical Outcomes Scale

    10 years

  • Epistaxis Characterization with Epistaxis Severity Scale

    10 years

  • Organ Vascular Malformation (VM) Development and Growth Assessment using Organ VMs Assessment Tool

    10 years

  • Treatment Outcomes Assessment with HHT Treatment Outcomes Scale

    10 years

Study Arms (1)

HHT patients

Those diagnosed with HHT based on the Curacao diagnostic criteria or genetic testing

Eligibility Criteria

Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

All patients evaluated and diagnosed with HHT in the U.S. will be eligible to participate in the study.

You may qualify if:

  • Diagnosed with HHT based on the Curacao diagnostic criteria or genetic testing.
  • Able to provide informed consent or informed consent via a parent or legally authorized representative due to their age or medical condition.

You may not qualify if:

  • Unable to provide informed consent or informed consent via a parent or legally authorized representative.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

RECRUITING

University of California, Los Angeles

Los Angeles, California, 90095, United States

RECRUITING

University of California, San Francisco

San Francisco, California, 94107, United States

RECRUITING

University of Colorado, Denver

Aurora, Colorado, 80045, United States

RECRUITING

Yale University

New Haven, Connecticut, 06520, United States

RECRUITING

Augusta University

Augusta, Georgia, 30912, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

Columbia University

New York, New York, 10032, United States

RECRUITING

University of North Carolina, Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

The Cleveland Clinic

Cleveland, Ohio, 44106, United States

RECRUITING

Oregon Health and Science University

Portland, Oregon, 97239, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75390, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84132, United States

RECRUITING

MeSH Terms

Conditions

Telangiectasia, Hereditary HemorrhagicArteriovenous MalformationsTelangiectasisEpistaxisGastrointestinal HemorrhageIntracranial Arteriovenous MalformationsVascular Malformations

Condition Hierarchy (Ancestors)

Hemostatic DisordersVascular DiseasesCardiovascular DiseasesHemorrhagic DisordersHematologic DiseasesHemic and Lymphatic DiseasesCardiovascular AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNose DiseasesRespiratory Tract DiseasesOtorhinolaryngologic DiseasesHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSigns and Symptoms, RespiratorySigns and SymptomsGastrointestinal DiseasesDigestive System DiseasesCerebrovascular DisordersBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCentral Nervous System Vascular MalformationsNervous System MalformationsIntracranial Arterial Diseases

Study Officials

  • Melissa A Dickey, MSN

    Cure HHT

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Melissa A Dickey, MSN

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
10 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 22, 2024

First Posted

February 14, 2024

Study Start

November 13, 2023

Primary Completion (Estimated)

November 1, 2033

Study Completion (Estimated)

November 1, 2033

Last Updated

June 13, 2025

Record last verified: 2025-06

Locations