Overview
Primary sclerosing cholangitis (PSC) is a chronic, progressive cholestatic liver disease characterized by inflammation and fibrosis of the intrahepatic and/or extrahepatic bile ducts. This process leads to multifocal bile duct strictures (narrowing) and eventually biliary cirrhosis, liver failure, and an increased risk of cholangiocarcinoma (bile duct cancer). PSC primarily affects the hepatobiliary system but is strongly associated with inflammatory bowel disease (IBD), particularly ulcerative colitis, which occurs in approximately 60–80% of PSC patients. Key symptoms include fatigue, pruritus (itching), jaundice, right upper quadrant abdominal pain, and recurrent episodes of bacterial cholangitis (bile duct infection) with fever and chills. As the disease progresses, patients may develop portal hypertension, hepatosplenomegaly, fat-soluble vitamin deficiencies, and metabolic bone disease. Laboratory findings typically show elevated alkaline phosphatase and gamma-glutamyl transferase levels. Diagnosis is usually established by magnetic resonance cholangiopancreatography (MRCP) or endoscopic retrograde cholangiopancreatography (ERCP), which reveal the characteristic beaded appearance of bile ducts due to alternating strictures and dilatations. There is currently no approved pharmacological therapy that has been proven to halt or reverse the progression of PSC. Ursodeoxycholic acid (UDCA) is commonly prescribed and may improve liver biochemistry, but its impact on long-term outcomes remains debated. Management is largely supportive, focusing on treatment of pruritus, nutritional supplementation, management of cholangitis episodes, endoscopic dilation of dominant strictures, and surveillance for cholangiocarcinoma and colorectal cancer in patients with concurrent IBD. Liver transplantation remains the only definitive treatment for end-stage PSC, though the disease can recur in the transplanted liver in approximately 20–25% of cases. PSC is more common in males, with a male-to-female ratio of approximately 2:1.
Also known as:
Clinical phenotype terms— hover any for plain English:
Multifactorial
Caused by a mix of several genes and environmental factors
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
10 eventsMassachusetts General Hospital
Assistance Publique - Hôpitaux de Paris — PHASE2
Ipsen — PHASE3
National Institute of Neurological Disorders and Stroke (NINDS) — PHASE1
Takeda — PHASE1
McMaster University — PHASE2
LISCure Biosciences — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Primary sclerosing cholangitis.
20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Primary sclerosing cholangitis.
Community
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Start the conversation →Latest news about Primary sclerosing cholangitis
Disease timeline:
New recruiting trial: Natural History of Post-Coronavirus Disease 19 Convalescence at the National Institutes of Health
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Magnetic Resonance Analysis of Neural Inflammatory Factors and External Stimulation
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Heat thErapy And mobiLity in COVID-19 Survivors
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Immune-mediated Pathogenic Mechanisms of Neuro-PASC in Veterans
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Osteopathic Manipulative Therapy Effects on Post-Acute Sequelae of COVID-19 (PASC) or Long COVID
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Phase 2/Phase 3 Study To Evaluate The Efficacy And Safety Of Ramatroban Along With The Standard Of Care In Subjects Hospitalized For COVID Pneumonia
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Brain Stimulation in Long COVID
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Low Dose Sirolimus in People With Post-Acute Sequelae of COVID-19 (PASC) Long COVID-19
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Sauna for Long Covid
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
New recruiting trial: Obesity, Insulin Resistance, and PASC: Persistent SARS-CoV-2
A new clinical trial is recruiting patients for Primary sclerosing cholangitis
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 Primary sclerosing cholangitis
What is Primary sclerosing cholangitis?
Primary sclerosing cholangitis (PSC) is a chronic, progressive cholestatic liver disease characterized by inflammation and fibrosis of the intrahepatic and/or extrahepatic bile ducts. This process leads to multifocal bile duct strictures (narrowing) and eventually biliary cirrhosis, liver failure, and an increased risk of cholangiocarcinoma (bile duct cancer). PSC primarily affects the hepatobiliary system but is strongly associated with inflammatory bowel disease (IBD), particularly ulcerative colitis, which occurs in approximately 60–80% of PSC patients. Key symptoms include fatigue, prurit
How is Primary sclerosing cholangitis inherited?
Primary sclerosing cholangitis follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Primary sclerosing cholangitis typically begin?
Typical onset of Primary sclerosing cholangitis is adult. Age of onset can vary across affected individuals.
Are there clinical trials for Primary sclerosing cholangitis?
Yes — 20 recruiting clinical trials are currently listed for Primary 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 Primary sclerosing cholangitis?
25 specialists and care centers treating Primary sclerosing cholangitis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.