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Active, not recruitingNCT03440697TAAUpdated Feb 13, 2026

Pathogenetic Basis of Aortopathy and Aortic Valve Disease

An observational study in Aortopathies, Thoracic Aortic Aneurysm and Aortic Valve Disease, sponsored by Yale University. Active, not recruiting at 2 sites in United States. Per ClinicalTrials.gov, last updated 2026-02-13.

Sponsored by Yale University · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
3,000
Sex
All
01

Study summary

The main purpose of this study is to define the complex genetic and pathogenic basis of thoracic aortic aneurysm (TAA) and other forms of aortopathy and/or aortic valve disease by identifying novel disease-causing genes and by identifying important genetic modifiers for aortic and aortic valve disease severity.

Read the detailed description

Thoracic aortic aneurysm (TAA) is a type of aortopathy describing dilation of the proximal aortic dimensions including the aortic root, which is a risk factor for aortic dissection and sudden cardiac death. TAA and other forms of aortopathy (e.g. aortic tortuosity or aortic hypoplasia/stenosis) develop in the presence or absence of additional cardiovascular malformations including bicuspid aortic valve. TAA is associated with connective tissue disorders (e.g. Marfan syndrome), and familial clustering has been identified in a significant proportion of nonsyndromic cases, establishing high heritability. Pedigree analysis of TAA kindreds clearly identifies complex inheritance; however, progress towards understanding the genetic basis of TAA and other forms of aortopathy and, ultimately, the susceptibility to aortic dissection remains incomplete. There is a clinical need to develop novel methods for predicting disease risk based on genotype and phenotype, to further elucidate the genetic and pathogenic mechanisms of aortopathy, and to improve medical and surgical therapies. The overarching hypothesis of this study is that individual genetic variation modulates susceptibility to disease severity and progression. The goals of this study are 1) to ascertain a cohort of subjects who have aortopathy and/or aortic valve disease including TAA or who have genetic risk for the development of aortopathy and/or aortic valve disease, 2) to collect paired blood and tissue samples from well-characterized subjects, family members of subjects, and controls to perform genome-wide DNA sequence, histopathologic, transcriptional, and proteomic analyses, and 3) to establish a tissue biorepository with detailed phenotype information to facilitate a broad spectrum of current and future studies.

02

Conditions studied

  • Aortopathies
  • Thoracic Aortic Aneurysm
  • Aortic Valve Disease
  • Thoracic Aortic Disease
  • Thoracic Aortic Dissection
  • Thoracic Aortic Rupture
  • Ascending Aortic Disease
  • Descending Aortic Disease
  • Ascending Aortic Aneurysm
  • Descending Aortic Aneurysm
  • Marfan Syndrome
  • Loeys-Dietz Syndrome
  • Vascular Ehlers-Danlos Syndrome
  • Shprintzen-Goldberg Syndrome
  • Turner Syndrome
  • PHACE Syndrome
  • Autosomal Recessive Cutis Laxa
  • Congenital Contractural Arachnodactyly
  • Arterial Tortuosity Syndrome
  • Bicuspid Aortic Valve-Associated Aortopathy
  • Bicuspid Aortic Valve
  • Familial Thoracic Aortic Aneurysm and Aortic Dissection

Keywords

  • Cardiac Disease
  • Cardiovascular Genetics
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Families affected by aortopathy, aortic valve disease, or syndromic or genetic diagnosis that poses risk for the development of aortic disease who have not yet developed disease.

Inclusion criteria

  • Open to external enrollment:

    • Subjects with a genetic diagnosis of Marfan Syndrome (MDS), Loeys-Dietz Syndrome (LDS), or Vascular Ehlers-Danlos Syndrome (EDS); (Positive genetic testing or a previous cardiac study required to be eligible)
    • Family members of eligible subjects (Only family members of subjects with syndromic diagnoses are eligible for external enrollment at this time)
  • Closed to external enrollment:

    • Subjects with aortic disease including TAA* or dissection, aortic tortuosity, or aortic hypoplasia/stenosis (based on any cardiac imaging modality including echocardiography, CT, MRI, or angiography)
    • Subjects with aortic valve disease (bicuspid, unicuspid, or tricuspid disease)
    • Control subjects having tissue removed during a surgical procedure (e.g. coronary artery bypass graft surgery (CABG), cardiac transplant, etc.)

Exclusion criteria

Exclusion Criteria:

  • Inability or unwillingness to provide consent (assent when indicated)
04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
3,000 participants (estimated)
Patient registry
No
Biospecimen retention
Samples with dna

Groups and cohorts

  • Aortopathy- Closed to external enrollment

    Subjects with aortic disease including TAA or dissection, aortic tortuosity, or aortic hypoplasia/stenosis (based on any cardiac imaging modality including echocardiography, CT, MRI, or angiography)

  • Syndromic- Open to external enrollment

    Subjects with a genetic diagnosis of Marfan Syndrome (MFS), Loeys-Dietz Syndrome (LDS), Vascular Ehlers-Danlos Syndrome (EDS) •positive genetic testing and/or a previous cardiac study required to be eligible

  • Aortopathy with Positive Genetic Results- Open to Enrollment

    Subjects with aortic disease including TAA or dissection, aortic tortuosity, or aortic hypoplasia/stenosis (based on any cardiac imaging modality including echocardiography, CT, MRI, or angiography) who also have positive genetic testing results related to aortopathy.

  • Aortic Valve Disease- Closed to enrollment

    Subjects with aortic valve disease (bicuspid, unicuspid, or tricuspid disease)

  • Family Members- Open to external enrollment

    Family members of eligible subjects •Only family members of subjects with syndromic diagnoses are eligible for external enrollment at this time

  • Controls- Closed to external enrollment

    Control subjects having tissue removed during a surgical procedure (e.g. coronary artery bypass graft surgery (CABG), cardiac transplant, etc.)

05

What researchers measure

Primary outcomes

  1. Biorepository Establishment

    Establish a biorepository with detailed phenotype information to facilitate a broad spectrum of current and future studies

    Time frame: 20 years

  2. Genetic Analysis

    The mechanisms of TAA pathogenesis will be determined by studying explanted aortic tissue and cells derived from patients with TAA for gene expression, protein expression, and other functional assays.

    Time frame: 20 years

06

Study locations

2 sites
  • Childrens Healthcare of Atlanta
    Atlanta, Georgia 30329, United States
  • IU School of Medicine
    Indianapolis, Indiana 46202, United States
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03440697
Lead sponsor
Yale University
Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Responsible party
Sponsor
First posted
Feb 22, 2018
Start date
Dec 10, 2015
Primary completion
Dec 31, 2030 (estimated)
Completion
Dec 31, 2030 (estimated)
Last update
Feb 13, 2026

Study contacts

Benjamin Landis, MD
principal investigator · Yale University

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is active, not recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.

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