Familial bicuspid aortic valve

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ORPHA:402075OMIM:109730Q23.1
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16Active trials12Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Familial bicuspid aortic valve (BAV) is an inherited heart condition in which the aortic valve — the valve that controls blood flow from the heart to the body's main artery (the aorta) — has two flaps (cusps) instead of the normal three. This is the most common congenital heart defect, and when it runs in families, it is called familial bicuspid aortic valve. Many people with a bicuspid aortic valve have no symptoms for years and may not know they have the condition until adulthood. However, over time the abnormal valve can lead to problems such as narrowing of the valve (aortic stenosis), leaking of the valve (aortic regurgitation), or enlargement (dilation) of the aorta, which increases the risk of a dangerous tear in the aortic wall (aortic dissection). Some people develop symptoms like chest pain, shortness of breath, dizziness, or fatigue, especially during exercise. In the familial form, multiple family members are affected, suggesting a strong genetic component. Treatment depends on how well the valve functions and ranges from regular monitoring with echocardiograms to medications that reduce strain on the heart, and in more severe cases, surgical repair or replacement of the aortic valve. Early detection through family screening is important because it allows doctors to monitor the heart and intervene before serious complications develop.

Also known as:

Key symptoms:

Heart murmur heard by a doctorShortness of breath, especially during exerciseChest pain or tightnessDizziness or faintingFatigue or feeling unusually tiredRapid or irregular heartbeat (palpitations)Difficulty exercising or reduced exercise toleranceSwelling in the ankles or feetNarrowing of the aortic valve (aortic stenosis)Leaking of the aortic valve (aortic regurgitation)Enlargement or widening of the aorta (aortic dilation)In severe cases, signs of heart failure

Clinical phenotype terms (12)— hover any for plain English
Aortic valve calcificationHP:0004380Thoracic aorta calcificationHP:0004962Aortic arch aneurysmHP:0005113Ascending aortic dissectionHP:0004933Abnormal left ventricular outflow tract morphologyHP:0011103
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Apr 2026TAVR vs SAVR in Severe Bicuspid Aortic Stenosis

Cedars-Sinai Medical Center — NA

TrialNOT YET RECRUITING
May 2025Towards a More Standardized Bicuspid Aortic Valve Repair: Rationale and Design of CONTOUR Trial - a Randomized Trial

University Hospital Augsburg — NA

TrialRECRUITING
Apr 2025Sapien 3 Transcatheter Aortic Valve Replacement in Young Aortic Valve Stenosis Patients From China

Xijing Hospital

TrialRECRUITING
Jan 2025Bicuspid Aortic Valve Replacement: Evaluation of Transcatheter Versus Surgery-Pilot Trial

Cedars-Sinai Medical Center — NA

TrialRECRUITING
Jan 2025A Study of Chinese Congenital Bicuspid Aortic Valve

Ruijin Hospital

TrialRECRUITING
Dec 2024Size Up Bicuspid with the LIra MEthod: the SUBLIME Study

IRCCS San Raffaele

TrialRECRUITING
Dec 2024Valvular Assessment of New Generation Aortic Replacement Devices

Instituto Dante Pazzanese de Cardiologia — NA

TrialRECRUITING
May 2024NavIIcusp: Bicuspid Aortic Valve Stenosis With Navitor Platform International Experience

Didier TCHETCHE

TrialNOT YET RECRUITING
Oct 2023Genetic Test Based Risk Prediction of Early Calcific Aortic Valve Disease in Patients With Bicuspid Aortic Valve

Yonsei University

TrialRECRUITING
Sep 2023Feasibility, Safety, and Effectiveness of the ACURATE neo2 Transcatheter Heart Valve for Severe Bicuspid Aortic Stenosis

National University of Ireland, Galway, Ireland

TrialENROLLING BY INVITATION

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Familial bicuspid aortic valve.

16 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

16 recruitingView all trials with filters →
N/A4 trials
Bicuspid Aortic Valve Replacement: Evaluation of Transcatheter Versus Surgery-Pilot Trial
N/A
Actively Recruiting
PI: Raj Makkar, MD (Cedars-Sinai Medical Center) · Sites: Los Angeles, California · Age: 5099 yrs
Medtronic Transcatheter Aortic Valve Replacement (TAVR) Low Risk Bicuspid Study
N/A
Active
PI: Michael Reardon, MD (The Methodist Hospital Research Institute) · Sites: Phoenix, Arizona; La Jolla, California +23 more · Age: 6099 yrs
Valvular Assessment of New Generation Aortic Replacement Devices
N/A
Actively Recruiting
· Sites: São Paulo, São Paulo · Age: 1865 yrs
Towards a More Standardized Bicuspid Aortic Valve Repair: Rationale and Design of CONTOUR Trial - a Randomized Trial
N/A
Actively Recruiting
· Sites: Augsburg, Bavaria; Frankfurt am Main, Hesse +2 more · Age: 1899 yrs
Other12 trials
Bicuspid Aortic Valve (BAV) Registry
Actively Recruiting
PI: Bo Yang, MD (University of Michigan) · Sites: Ann Arbor, Michigan · Age: 1899 yrs
A Study of Chinese Congenital Bicuspid Aortic Valve
Actively Recruiting
PI: Zhengbin Zhu, MD, PhD (Ruijin Hospital) · Sites: Shanghai, Shanghai Municipality · Age: 1460 yrs
International Bicuspid Aortic Valve Consortium (BAVCon)
Active
PI: Simon C. Body, MD, MPH (Boston University School of Medicine, Anesthesiolo) · Sites: Boston, Massachusetts; Boston, Massachusetts +10 more · Age: 890 yrs
Sapien 3 Transcatheter Aortic Valve Replacement in Young Aortic Valve Stenosis Patients From China
Actively Recruiting
PI: Jian Yang, MD, PhD (Department of Cardiovascular Surgery of Xijing Hos) · Sites: Xi'an, Shaanxi; Chengdu, Sichuan +2 more · Age: 5070 yrs
Bicuspid Aortic Valve Stenosis Transcatheter Aortic Valve Replacement Registry
Actively Recruiting
· Sites: Los Angeles, California · Age: 19129 yrs
Identification of Genetic Causes of Bicuspid Aortic Valve Disease
Active
PI: Simon C Body, MD MPH (Boston University, Anesthesiology Department) · Sites: Boston, Massachusetts · Age: 1480 yrs
The BRAVE Study- The Identification of Genetic Variants Associated With Bicuspid Aortic Valve Using a Combination of Case-control and Family-based Approaches.
Actively Recruiting
PI: Aidan Bolger, Dr (Principal Investigator) · Sites: Kettering; Leicester +2 more · Age: 1099 yrs
Identification of Genetic Causes of Calcific Aortic Valve Disease
Active
· Sites: Boston, Massachusetts · Age: 2099 yrs
Genetic Test Based Risk Prediction of Early Calcific Aortic Valve Disease in Patients With Bicuspid Aortic Valve
Actively Recruiting
PI: Iksung Cho (Division of Cardiology, Severance Cardiovascular H) · Sites: Seoul · Age: 1980 yrs
Feasibility, Safety, and Effectiveness of the ACURATE neo2 Transcatheter Heart Valve for Severe Bicuspid Aortic Stenosis
Enrolling by Invitation
PI: Darren Mylotte, MD, PhD (University of Galway, Galway University Hospital) · Sites: Bad Nauheim; Galway +1 more · Age: 1899 yrs
Size Up Bicuspid with the LIra MEthod: the SUBLIME Study
Actively Recruiting
PI: Prof. Matteo Montorfano, MD (IRCCS San Raffaele Scientific Institute) · Sites: Brescia, Italy; Brescia, Italy +3 more · Age: 1899 yrs
Changes of Ascending Aortic Diameter in Patients Undergoing Transcatheter Aortic Valve Replacement
Actively Recruiting
· Sites: Beijing, Beijing Municipality

Specialists

12 foundView all specialists →
FM
Federico M. Asch, MD,FACC,FASE
WASHINGTON, DC
Specialist
PI on 1 active trial
RP
Reed E. Pyeritz, M.D., Ph.D.
PHILADELPHIA, PA
Specialist
PI on 1 active trial
DP
Dianna M. Milewicz, M.D. Ph.D.
HOUSTON, TX
Specialist
PI on 1 active trial
AD
Aidan Bolger, Dr
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Familial bicuspid aortic valve.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Familial bicuspid aortic valve

Disease timeline:

New recruiting trial: The BRAVE Study- The Identification of Genetic Variants Associated With Bicuspid Aortic Valve Using a Combination of Case-control and Family-based Approaches.

A new clinical trial is recruiting patients for Familial bicuspid aortic valve

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.How well is my aortic valve currently functioning, and is there any narrowing or leaking?,Is my aorta enlarged, and how often should it be monitored?,Are there any activities or sports I should avoid?,Should my family members (parents, siblings, children) be screened with an echocardiogram?,Would genetic testing be helpful for my family?,At what point would surgery be recommended, and what type of surgery would be best for me?,Do I need to take any medications now, and do I need antibiotics before dental procedures?

Common questions about Familial bicuspid aortic valve

What is Familial bicuspid aortic valve?

Familial bicuspid aortic valve (BAV) is an inherited heart condition in which the aortic valve — the valve that controls blood flow from the heart to the body's main artery (the aorta) — has two flaps (cusps) instead of the normal three. This is the most common congenital heart defect, and when it runs in families, it is called familial bicuspid aortic valve. Many people with a bicuspid aortic valve have no symptoms for years and may not know they have the condition until adulthood. However, over time the abnormal valve can lead to problems such as narrowing of the valve (aortic stenosis), lea

How is Familial bicuspid aortic valve inherited?

Familial bicuspid aortic valve follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for Familial bicuspid aortic valve?

Yes — 16 recruiting clinical trials are currently listed for Familial bicuspid aortic valve on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Familial bicuspid aortic valve?

12 specialists and care centers treating Familial bicuspid aortic valve are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.