Trial Title:
Comprehensive HHT Outcomes Registry of the United States (CHORUS)
NCT ID:
NCT06259292
Condition:
Hereditary Hemorrhagic Telangiectasia
Arteriovenous Malformations
Telangiectasia
Epistaxis
GastroIntestinal Bleeding
Cerebral Arteriovenous Malformations
Vascular Malformation
Conditions: Official terms:
Hemangioma
Gastrointestinal Hemorrhage
Epistaxis
Intracranial Arteriovenous Malformations
Telangiectasis
Arteriovenous Malformations
Telangiectasia, Hereditary Hemorrhagic
Vascular Malformations
Congenital Abnormalities
Study type:
Observational [Patient Registry]
Overall status:
Recruiting
Study design:
Time perspective:
Prospective
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.
Detailed description:
The Comprehensive HHT Outcomes Registry of the United States (CHORUS) is a research
initiative led by the HHT Foundation International, Inc. ("Cure HHT"). The study focuses
on Hereditary Hemorrhagic Telangiectasia (HHT), a rare genetic disorder characterized by
the development of abnormal blood vessels in various organs of the body, including the
brain, spine, lungs, liver, GI tract, skin, nasal mucosa, and oral cavity. The prevalence
of HHT is estimated to be 1 in 5000, affecting children and adults. These abnormal blood
vessels can lead to acute and chronic bleeding, stroke, heart failure, and death.
Treatments are currently predominately limited to managing complications, while
approximately 90% of adults have ongoing symptoms, despite the best surgical and medical
therapies. With recent drug developments related to angiogenesis, there is hope for
effective novel therapies.
A natural history registry for HHT has significant implications for improving the
understanding and management of this rare genetic disorder. The purpose of this study is
to better understand HHT, the symptoms and complications it causes ("outcomes"), and to
understand how the disease impacts people's lives. The investigators hypothesize that a
natural history registry for HHT will improve our understanding of the disease, lead to
better management of patients, and ultimately, contribute to developing novel therapies
to treat this disease.
The investigators will collaborate with multiple HHT Centers of Excellence across the
U.S. to establish a comprehensive registry of HHT patients. The study aims to enroll
approximately 10,000 HHT patients over a 10-year period. Longitudinal data will be
collected both retrospectively and prospectively, with a focus on increasing the
understanding of this rare disease, accelerating the development of new diagnostic and
treatment options, and working collaboratively with clinicians who care for individuals
with HHT to identify and address gaps in the system of care, especially those from
underserved populations.
The study aims to serve as a centralized resource for future clinical trials and research
in HHT. Data security and confidentiality are prioritized, and participants have the
option to withdraw from the study at any time. The study is funded by the U.S. Department
of Health Resources and Service Administration (HRSA) through a grant awarded to Cure
HHT.
Criteria for eligibility:
Study pop:
All patients evaluated and diagnosed with HHT in the U.S. will be eligible to participate
in the study.
Sampling method:
Non-Probability Sample
Criteria:
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Unable to provide informed consent or informed consent via a parent or legally
authorized representative.
Gender:
All
Minimum age:
N/A
Maximum age:
N/A
Healthy volunteers:
Accepts Healthy Volunteers
Locations:
Facility:
Name:
University of California, Los Angeles
Address:
City:
Los Angeles
Zip:
90095
Country:
United States
Status:
Recruiting
Contact:
Last name:
Sayeh Jafari
Phone:
424-467-5809
Email:
SayehJafari@mednet.ucla.edu
Contact backup:
Last name:
Victoria Rueda
Phone:
310-562-9694
Email:
vrueda@mednet.ucla.edu
Investigator:
Last name:
Justin McWilliams, MD
Email:
Principal Investigator
Investigator:
Last name:
Lucas Cusumano, MD
Email:
Sub-Investigator
Facility:
Name:
University of California, San Francisco
Address:
City:
San Francisco
Zip:
94107
Country:
United States
Status:
Recruiting
Contact:
Last name:
Bridget Kilbride
Phone:
360-931-8439
Email:
bridget.kilbride@ucsf.edu
Investigator:
Last name:
Steven Hetts, MD
Email:
Principal Investigator
Investigator:
Last name:
Miles Conrad, MD
Email:
Sub-Investigator
Facility:
Name:
University of Colorado, Denver
Address:
City:
Aurora
Zip:
80045
Country:
United States
Status:
Recruiting
Contact:
Last name:
Johan Allingmon
Phone:
303-724-6052
Email:
johan.allingmon@cuanschutz.edu
Investigator:
Last name:
Peter Hountras, MD
Email:
Principal Investigator
Investigator:
Last name:
Todd Bull, MD
Email:
Sub-Investigator
Facility:
Name:
Yale University
Address:
City:
New Haven
Zip:
06520
Country:
United States
Status:
Recruiting
Contact:
Last name:
Katharine Henderson
Phone:
203-737-1427
Email:
katharine.henderson@yale.edu
Investigator:
Last name:
Jeffrey Pollak, MD
Email:
Principal Investigator
Facility:
Name:
Augusta University
Address:
City:
Augusta
Zip:
30912
Country:
United States
Status:
Recruiting
Contact:
Last name:
Melissa James, RN
Phone:
706-721-5599
Email:
mejames@augusta.edu
Investigator:
Last name:
James Gossage, MD
Email:
Principal Investigator
Facility:
Name:
Massachusetts General Hospital
Address:
City:
Boston
Zip:
02114
Country:
United States
Status:
Recruiting
Contact:
Last name:
Madeleine Macy, BS
Phone:
617-724-0536
Email:
mmacy1@mgh.harvard.edu
Investigator:
Last name:
Josanna Rodriguez-Lopez, MD
Email:
Principal Investigator
Investigator:
Last name:
Allison Witkin, MD
Email:
Sub-Investigator
Investigator:
Last name:
Alexandra Wong, MD
Email:
Sub-Investigator
Facility:
Name:
Mayo Clinic
Address:
City:
Rochester
Zip:
55905
Country:
United States
Status:
Recruiting
Contact:
Last name:
Greg Schwichtenberg
Phone:
507-255-1245
Email:
Schwichtenberg.Greg@mayo.edu
Contact backup:
Last name:
Sue Ann Donlinger
Phone:
507-284-9259
Email:
Donlinger.SueAnn@mayo.edu
Investigator:
Last name:
Vivek Iyer, MD
Email:
Principal Investigator
Facility:
Name:
Washington University School of Medicine
Address:
City:
Saint Louis
Zip:
63110
Country:
United States
Status:
Recruiting
Contact:
Last name:
Melissa Beasley, AA
Phone:
314-273-5922
Email:
beasleym@wustl.edu
Contact backup:
Last name:
Kristine Kempf
Phone:
314-273-8131
Email:
kempf@wustl.edu
Investigator:
Last name:
Murali Chakinala, MD
Email:
Principal Investigator
Facility:
Name:
Columbia University
Address:
City:
New York
Zip:
10032
Country:
United States
Status:
Recruiting
Contact:
Last name:
Kelsey Weddig, RN, BSN
Phone:
212-305-7470
Email:
kw3149@cumc.columbia.edu
Investigator:
Last name:
Charles Murphy, MD
Email:
Principal Investigator
Facility:
Name:
University of North Carolina, Chapel Hill
Address:
City:
Chapel Hill
Zip:
27599
Country:
United States
Status:
Recruiting
Contact:
Last name:
Karen Smith, RN
Phone:
916-966-2790
Email:
karens@med.unc.edu
Contact backup:
Last name:
Kristi Kirkland
Phone:
919-966-2790
Email:
kristi_kirkland@med.unc.edu
Investigator:
Last name:
Raj Kasthuri, MBBS
Email:
Principal Investigator
Facility:
Name:
The Cleveland Clinic
Address:
City:
Cleveland
Zip:
44106
Country:
United States
Status:
Recruiting
Contact:
Last name:
Katherine Miyoshi
Phone:
216-554-9393
Email:
miyoshk@ccf.org
Investigator:
Last name:
Joseph Parambil, MD
Email:
Principal Investigator
Investigator:
Last name:
Keith McCrae, MD
Email:
Sub-Investigator
Investigator:
Last name:
John Carl, MD
Email:
Sub-Investigator
Facility:
Name:
Oregon Health and Science University
Address:
City:
Portland
Zip:
97239
Country:
United States
Status:
Recruiting
Contact:
Last name:
Lori Russell, RN
Phone:
503-494-7226
Email:
watsonlo@ohsu.edu
Contact backup:
Last name:
Eleanor Lottsfeldt, BA
Phone:
503-494-3199
Email:
lottsfel@ohsu.edu
Investigator:
Last name:
Mark Chesnutt, MD
Email:
Principal Investigator
Investigator:
Last name:
Claire Kaufman, MD
Email:
Sub-Investigator
Facility:
Name:
University of Pennsylvania
Address:
City:
Philadelphia
Zip:
19104
Country:
United States
Status:
Recruiting
Contact:
Last name:
Ala Streater, BS
Phone:
215-662-3622
Email:
ala.streater@pennmedicine.upenn.edu
Contact backup:
Last name:
Isaac Elysee, MS
Phone:
215-614-0833
Email:
isaac.elysee@pennmedicine.upenn.edu
Investigator:
Last name:
Theodore Drivas, MD
Email:
Principal Investigator
Facility:
Name:
University of Texas Southwestern
Address:
City:
Dallas
Zip:
75390
Country:
United States
Status:
Recruiting
Contact:
Last name:
Ahmad Ara'r, MD
Phone:
682-418-7730
Email:
ahmad.arar@utsouthwestern.edu
Investigator:
Last name:
An Lu, MD
Email:
Principal Investigator
Investigator:
Last name:
John Battaile, MD
Email:
Sub-Investigator
Facility:
Name:
University of Utah
Address:
City:
Salt Lake City
Zip:
84132
Country:
United States
Status:
Recruiting
Contact:
Last name:
Cassidy Sion, RN, BSN
Phone:
801-581-8188
Email:
u0828999@umail.utah.edu
Investigator:
Last name:
Kevin Whitehead, MD
Email:
Principal Investigator
Start date:
November 13, 2023
Completion date:
November 2033
Lead sponsor:
Agency:
Cure HHT
Agency class:
Other
Collaborator:
Agency:
Augusta University
Agency class:
Other
Collaborator:
Agency:
The Cleveland Clinic
Agency class:
Other
Collaborator:
Agency:
Mayo Clinic
Agency class:
Other
Collaborator:
Agency:
Massachusetts General Hospital
Agency class:
Other
Collaborator:
Agency:
Columbia University
Agency class:
Other
Collaborator:
Agency:
Oregon Health and Science University
Agency class:
Other
Collaborator:
Agency:
University of California, Los Angeles
Agency class:
Other
Collaborator:
Agency:
University of California, San Francisco
Agency class:
Other
Collaborator:
Agency:
University of Colorado, Denver
Agency class:
Other
Collaborator:
Agency:
University of North Carolina, Chapel Hill
Agency class:
Other
Collaborator:
Agency:
University of Pennsylvania
Agency class:
Other
Collaborator:
Agency:
University of Texas
Agency class:
Other
Collaborator:
Agency:
University of Utah
Agency class:
Other
Collaborator:
Agency:
Washington University School of Medicine
Agency class:
Other
Collaborator:
Agency:
Yale University
Agency class:
Other
Collaborator:
Agency:
Health Resources and Services Administration (HRSA)
Agency class:
U.S. Fed
Collaborator:
Agency:
Children's Hospital of Philadelphia
Agency class:
Other
Source:
Cure HHT
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06259292
