Sclerosing cholangitis

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ORPHA:447771
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5Active trials40Specialists8Treatment centers

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Overview

Sclerosing cholangitis is a serious liver disease that affects the bile ducts — the tubes that carry bile from the liver to the small intestine. In this condition, the bile ducts become inflamed and scarred over time, causing them to narrow and eventually block the flow of bile. When bile cannot drain properly, it builds up in the liver and causes damage. There are two main forms: primary sclerosing cholangitis (PSC), which is the most common type and is often linked to inflammatory bowel disease, and secondary sclerosing cholangitis, which is caused by a known trigger such as infection, surgery, or injury to the bile ducts. Over time, the scarring and blockage can lead to serious complications including liver cirrhosis (severe scarring of the liver), liver failure, and an increased risk of bile duct cancer (cholangiocarcinoma). Common symptoms include fatigue, itching, yellowing of the skin and eyes (jaundice), abdominal pain, and recurring infections of the bile ducts called cholangitis episodes. There is currently no medication proven to stop or reverse the disease. Treatment focuses on managing symptoms, preventing complications, and treating infections. In advanced cases, a liver transplant may be the only life-saving option. Research into new therapies is ongoing, and patients are encouraged to be followed by a specialist experienced in liver disease.

Key symptoms:

Persistent fatigue and low energyItching of the skin (pruritus), often severeYellowing of the skin and whites of the eyes (jaundice)Pain or discomfort in the upper right abdomenFever and chills, especially during bile duct infectionsDark urinePale or greasy stoolsUnintended weight lossSwelling of the abdomen due to fluid buildup (ascites)Easy bruising or bleedingBone pain or increased risk of fractures due to poor vitamin absorptionRecurring episodes of bacterial infection in the bile ducts (cholangitis)

Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

5 events
Nov 2025DEFINING THE GENETIC DRIVERS OF ADULT-ONSET CHOLESTATIC LIVER DISEASE

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico — NA

TrialRECRUITING
Aug 2025Evaluate PK & Safety of Saroglitazar in Subjects With Moderate Hepatic Impairment Due to Cholestatic Liver Disease

Zydus Therapeutics Inc. — PHASE1

TrialRECRUITING
Jan 2024Genotype-phenotype Relationship Between Cryptogenic Cholestasis and Familial Intrahepatic Cholestasis

IRCCS Azienda Ospedaliero-Universitaria di Bologna

TrialRECRUITING
Dec 2015Secondary Sclerosing Cholangitis in Critically Ill Patients

Medical University of Graz

TrialACTIVE NOT RECRUITING
Aug 2015The Necessity of Bile Cultures in Patients With Acute Cholangitis

Seoul National University Hospital — NA

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Sclerosing cholangitis.

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

View clinical trials →

Clinical Trials

5 recruitingView all trials with filters →
Phase 11 trial
Evaluate PK & Safety of Saroglitazar in Subjects With Moderate Hepatic Impairment Due to Cholestatic Liver Disease
Phase 1
Actively Recruiting
PI: Deven Parmar (Zydus Therapeutics Inc.) · Sites: Indianapolis, Indiana · Age: 1880 yrs
N/A2 trials
The Necessity of Bile Cultures in Patients With Acute Cholangitis
N/A
Active
PI: Sang Hyub Lee, MD. PhD. (Department of internal medicine and liver research) · Sites: Seoul · Age: 2099 yrs
DEFINING THE GENETIC DRIVERS OF ADULT-ONSET CHOLESTATIC LIVER DISEASE
N/A
Actively Recruiting
PI: Luisa Ronzoni, Doctor · Sites: Milan, Milano · Age: 1865 yrs
Other2 trials
Genotype-phenotype Relationship Between Cryptogenic Cholestasis and Familial Intrahepatic Cholestasis
Actively Recruiting
PI: Giovanni Vitale, MD (IRCCS Azienda Ospedaliero-Universitaria di Bologna) · Sites: Bologna, Bologna; Modena, Modena · Age: 1899 yrs
Secondary Sclerosing Cholangitis in Critically Ill Patients
Active
PI: Vanessa Stadlbauer, MD (Medical University of Graz) · Sites: Graz · Age: 1899 yrs

Specialists

Showing 25 of 40View all specialists →
TF
Trine Folseraas
Specialist
4 Sclerosing cholangitis publications
CB
Christopher L Bowlus
SACRAMENTO, CA
Specialist
2 Sclerosing cholangitis publications
CP
Cyriel Ponsioen
Specialist
2 Sclerosing cholangitis publications
NC
Nora Cazzagon
Specialist
2 Sclerosing cholangitis publications
DA
David N Assis
NEW HAVEN, CT
Specialist
2 Sclerosing cholangitis publications
LF
Lisa Forman
Specialist
2 Sclerosing cholangitis publications
TK
Tom H Karlsen
Specialist
6 Sclerosing cholangitis publications
AB
Annika Bergquist
Specialist
5 Sclerosing cholangitis publications
JH
Johannes R Hov
Specialist
3 Sclerosing cholangitis publications
CS
Christoph Schramm
BRONX, NY
Specialist
3 Sclerosing cholangitis publications
FS
Francesca Saffioti
Specialist
2 Sclerosing cholangitis publications
JB
Jesus M Banales
Specialist
2 Sclerosing cholangitis publications
MT
Michael Trauner
OXNARD, CA
Specialist
2 Sclerosing cholangitis publications
AP
Annika Bergquist, MD PhD
Specialist
PI on 1 active trial
CM
Cynthia Levy, MD
Specialist
PI on 4 active trials
MP
Michael Trauner, Prof
OXNARD, CA
Specialist
PI on 1 active trial
MP
Michael P Manns, Prof
GRESHAM, OR
Specialist
PI on 1 active trial
RD
Rong Deng
NEW YORK, NY
Specialist
PI on 2 active trials14 Sclerosing cholangitis publications
KM
Kenneth Cox, MD
Specialist
PI on 1 active trial
MM
Mark D Topazian, MD
ROCHESTER, MN
Specialist
PI on 1 active trial
CJ
Channa Jayasekera
SCOTTSDALE, AZ
Specialist
PI on 1 active trial
AM
Alexander Miethke, MD
CINCINNATI, OH
Specialist
PI on 2 active trials1 Sclerosing cholangitis publication
SP
Stephen J Rossi, PharmD
Specialist
PI on 3 active trials
AM
Amy E Taylor, MD
Specialist
PI on 1 active trial1 Sclerosing cholangitis publication

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 Sclerosing cholangitis.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Sclerosing cholangitis

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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.

Questions for your doctor

Bring these to your next appointment

  • Q1.What stage is my liver disease at, and how quickly might it progress?,How often do I need imaging and blood tests to monitor my bile ducts and liver?,Should I be screened for bile duct cancer, and how is that done?,Are there any clinical trials I might be eligible for?,Do I need to be evaluated for a liver transplant now or in the future?,What symptoms should prompt me to go to the emergency room immediately?,Should I be tested for inflammatory bowel disease if I haven't been already?

Common questions about Sclerosing cholangitis

What is Sclerosing cholangitis?

Sclerosing cholangitis is a serious liver disease that affects the bile ducts — the tubes that carry bile from the liver to the small intestine. In this condition, the bile ducts become inflamed and scarred over time, causing them to narrow and eventually block the flow of bile. When bile cannot drain properly, it builds up in the liver and causes damage. There are two main forms: primary sclerosing cholangitis (PSC), which is the most common type and is often linked to inflammatory bowel disease, and secondary sclerosing cholangitis, which is caused by a known trigger such as infection, surge

How is Sclerosing cholangitis inherited?

Sclerosing cholangitis follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for Sclerosing cholangitis?

Yes — 5 recruiting clinical trials are currently listed for Sclerosing cholangitis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Sclerosing cholangitis?

25 specialists and care centers treating Sclerosing cholangitis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.