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NCT06297993Recruiting

Global Prospective, Observational Cohort of Adult Patients With Primary Sclerosing Cholangitis (WIND-PSC Study)

PSC Partners Seeking a Cure

Start Date

5/6/2024

Completion Date

1/1/2031

Summary

Develop an appropriate real-world data comparator cohort to support the design, execution, and serve as an external control for interventional clinical trials in PSC.

Detailed Description

Global, multi-center longitudinal observational cohort study. Collection of prospective clinical data to include liver-related clinical outcomes and safety events, hepatobiliary malignancies, relevant key biomarkers, imaging assessments, PSC-related clinical symptoms, patient-reported outcomes, and medication use in adult patients with PSC.

Eligibility Criteria

Age Range: 18 years to 75 years

Inclusion Criteria: 1. Adult patients between 18 and 75 years of age (inclusive) who can comprehend instructions, follow the study procedures and are willing to sign an Informed Consent Form (ICF). 2. Confirmed clinical diagnosis of large duct PSC. Exclusion Criteria: 1. Clinically significant acute or chronic liver disease of an etiology other than PSC (including but not limited to metabolic-dysfunction associated steatohepatitis (MASH), PBC, HCV, HBV, or alcoholic hepatitis, Wilson's disease, alpha-1 antitryp-sin deficiency, acute or chronic drug-induced liver injury) * Patients with PSC and elements of AIH overlap are allowed to enroll * Patients with metabolic dysfunction associated steatotic liver disease (MASLD) or benign steatosis are allowed to enroll 2. Small-Duct PSC. 3. Clinically diagnosed secondary or IgG4-related sclerosing cholangitis. 4. Clinically diagnosed infections (including acute cholangitis) and receiving treatment within the past 7 days; patients on chronic suppressive antibiotics for acute cholangitis will be allowed to enroll 5. Hospitalization in the past 7 days 6. UDCA dose \>28 mg/kg 7. Evidence of current or historical decompensated cirrhosis based on the following clinical events: * Ascites \> Grade 2 and requiring treatment * Esophageal or gastric variceal bleeding requiring hospitalization * Hepatic encephalopathy (as defined by a West Haven score ≥ 2) * Spontaneous bacterial peritonitis defined as ascites absolute neutrophil count \>250/mm3 in the absence of an intra-abdominal source of infection * AKI-HRS according to AASLD Guidelines (Flamm 2021) * Portal hypertension based on a platelet count \< 150 × 109/L and LSM \> 15 kPa with clinical, laboratory, imaging and/or other relevant parameters 8. Prior liver transplantation 9. MELD 3.0 Score \>15. For subjects on anticoagulation medication, baseline INR determination for MELD score calculation should take this use into account. 10. History, evidence, or high suspicion of hepatobiliary malignancies or active colon cancer based on imaging, screening laboratory values, and/or clinical symptoms. Patients with a history of colon cancer who have undergone a colectomy and have no current evidence of colon cancer will be allowed to enroll in the study. 11. Participants with current clinical or laboratory evidence of any severe, progressive, or uncontrolled disease, related or unrelated to PSC and which, in the opinion of the investigator, has an expected survival of less than 52 weeks. 12. Participants who are impaired, incapacitated, or incapable of completing study-related assessments or giving informed consent. 13. Prisoners or participants who are involuntarily incarcerated. 14. Participants who are currently participating in an investigational PSC therapy clinical study or who have participated in such a study within the past 12 weeks 15. Absence of data in medical records to assess inclusion and exclusion criteria.

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Conditions

PSC

Locations

UC Davis

Sacramento, California 95817

United States

California Pacific Medical Center

San Francisco, California 94109

United States

University of California, San Francisco

San Francisco, California 94143

United States

Yale University

New Haven, Connecticut 06510

United States

Schiff Center for Liver Diseases / University of Miami

Miami, Florida 33136

United States

Indiana University

Indianapolis, Indiana 46202

United States

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

Beth Israel Deaconess Medical Center

Boston, Massachusetts 02215

United States

Mayo Clinic

Rochester, Minnesota 55901

United States

UT Southwestern Medical Center

Dallas, Texas 75390

United States

Virginia Commonwealth University

Richmond, Virginia 23298

United States

University of Alberta

Edmonton, Alberta T6G 2X8

Canada

University Health Network

Toronto, Ontario M5G2C4

Canada

Centre de recherche du centre hospitalier de l'Université de Montréal (CRCHUM)

Montreal, Quebec H2X 0A9

Canada

University Medical Center Hamburg-Eppendorf

Hamburg, Free and Hanseatic City of Hamburg 20246

Germany

University of Milano Bicocca

Milan, Monza (MB) 20900

Italy

Auckland University

Auckland,

New Zealand

Oxford University Hospitals

Oxford, OX3 9DU

United Kingdom