Fontan-associated liver disease

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ORPHA:699068
Who is this for?
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4Active trials22Specialists8Treatment centers

Where are you in your journey?

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

Also known as:

Orphanet ↗NORD ↗

FDA & Trial Timeline

4 events
Aug 2025Cholate Clearance in Fontan and Heart Failure

HepQuant, LLC

TrialRECRUITING
Mar 2024IMProving DRug Dosing and Outcomes for Single VEntricle Patients With Fontan Associated Liver Disease

Children's Mercy Hospital Kansas City — PHASE1

TrialRECRUITING
Sep 2022Fontan Associated Liver Disease and the Evaluation of Biomarkers for Disease Severity Assessment

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

TrialRECRUITING
Jan 2018Fontan Associated Liver Disease

Children's Hospital Medical Center, Cincinnati

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Fontan-associated liver disease.

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

View clinical trials →

Clinical Trials

4 recruitingView all trials with filters →
Phase 11 trial
IMProving DRug Dosing and Outcomes for Single VEntricle Patients With Fontan Associated Liver Disease
Phase 1
Actively Recruiting
PI: Jonathan Wagner, DO (Children's Mercy) · Sites: Kansas City, Missouri · Age: 899 yrs
Other3 trials
Fontan Associated Liver Disease and the Evaluation of Biomarkers for Disease Severity Assessment
Actively Recruiting
PI: Theo Heller, M.D. (National Institute of Diabetes and Digestive and K) · Sites: Bethesda, Maryland · Age: 18100 yrs
Fontan Associated Liver Disease
Active
· Sites: Cincinnati, Ohio · Age: 05 yrs
Cholate Clearance in Fontan and Heart Failure
Actively Recruiting
· Sites: Philadelphia, Pennsylvania · Age: 1899 yrs

Specialists

22 foundView all specialists →
MH
Moira B Hilscher
ROCHESTER, MN
Specialist
3 Fontan-associated liver disease publications
JR
Jack Rychik
PHILADELPHIA, PA
Specialist
1 Fontan-associated liver disease publication
JZ
Juanjuan Zhao
Specialist
1 Fontan-associated liver disease publication
HB
Huajun Bai
Specialist
1 Fontan-associated liver disease publication
AB
Aidan Bauer
Specialist
1 Fontan-associated liver disease publication
WY
Wenbao Yu
Specialist
1 Fontan-associated liver disease publication
KD
Kathryn M Dodds
Specialist
1 Fontan-associated liver disease publication
SV
Sudhakar K Venkatesh
ROCHESTER, MN
Specialist
2 Fontan-associated liver disease publications
PK
Patrick S Kamath
ROCHESTER, MN
Specialist
3 Fontan-associated liver disease publications
ER
Elizabeth B Rand
Specialist
2 Fontan-associated liver disease publications
MW
Michael L Wells
Specialist
2 Fontan-associated liver disease publications
LT
Luis Téllez
Specialist
2 Fontan-associated liver disease publications
ES
Enrique Rodríguez de Santiago
Specialist
2 Fontan-associated liver disease publications
AA
Agustín Albillos
Specialist
2 Fontan-associated liver disease publications
PH
Po Hu
Specialist
1 Fontan-associated liver disease publication
TM
Theo Heller, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 8 active trials
DM
David J Goldberg, MD
Specialist
PI on 2 active trials
AE
Alexander C Egbe
ROCHESTER, MN
Specialist
PI on 1 active trial116 Fontan-associated liver disease publications
KM
Kurt R Schumacher, MD
ANN ARBOR, MI
Specialist
PI on 1 active trial
JD
Jonathan Wagner, DO
Specialist
PI on 1 active trial11 Fontan-associated liver disease publications
RM
Rahul Rathod, MD
BOSTON, MA
Specialist
PI on 2 active trials
MM
Miriam Michel, MD
LOS ANGELES, CA
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 Fontan-associated liver disease.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Fontan-associated liver disease

Disease timeline:

New recruiting trial: Fontan Associated Liver Disease and the Evaluation of Biomarkers for Disease Severity Assessment

A new clinical trial is recruiting patients for Fontan-associated liver disease

New recruiting trial: IMProving DRug Dosing and Outcomes for Single VEntricle Patients With Fontan Associated Liver Disease

A new clinical trial is recruiting patients for Fontan-associated liver disease

New recruiting trial: Cholate Clearance in Fontan and Heart Failure

A new clinical trial is recruiting patients for Fontan-associated liver disease

Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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.

Common questions about Fontan-associated liver disease

Are there clinical trials for Fontan-associated liver disease?

Yes — 4 recruiting clinical trials are currently listed for Fontan-associated liver disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Fontan-associated liver disease?

22 specialists and care centers treating Fontan-associated liver disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.