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

Recruiting

• Conditions: PSC

• Registration Number: NCT06297993
• Lead Sponsor: PSC Partners Seeking a Cure

Brief 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.

Study Timeline

• Study First Submitted: 2024-01-29
• Study First Submitted QC: 2024-02-29
• Study First Posted: 2024-03-07
• Study Start: 2024-05-06
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-22
• Last Update Posted: 2025-04-25
• Primary Completion: 2029-01
• Study Completion: 2031-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 2000

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 based on current AASLD Guidelines (Bowlus 2023).

Exclusion Criteria

1. Clinically significant acute or chronic liver disease of an etiology other than PSC (with or without presence of features of AIH).

2. Small-Duct PSC.

3. Clinically diagnosed secondary or IgG4-related sclerosing cholangitis.

4. Clinically diagnosed acute cholangitis and currently receiving treatment. Patients on chronic suppressive antibiotics will be allowed to enroll.

5. UDCA dose >28 mg/kg

6. 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)

7. Prior liver transplantation

8. MELD-Na Score >15. For subjects on anticoagulation medication, baseline INR determination for MELD score calculation should take this use into account.

9. 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 48 weeks.

10. Participants who are impaired, incapacitated, or incapable of completing study- related assessments or giving informed consent.

11. Prisoners or participants who are involuntarily incarcerated.

12. Participants who are currently participating in an interventional clinical study.

13. Absence of data in medical records to assess inclusion and exclusion criteria.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Develop interventional clinical trial external control using clinical and biomarker data.	Quarterly for five years from enrollment	Collection of liver-related data at enrollment and each quarterly and annual visit.

Secondary Outcome Measures

Name	Time	Method
Identify alternative endpoints to predict clinical benefit using large clinical and biomarker data.	Quarterly for five years from enrollment	Collection of biomarker and imaging at enrollment and each annual visit.
Changes in patient reported outcomes over time as assessed by Patient Reported Outcomes surveys.	Quarterly for five years from enrollment	Patient Reported Outcomes surveys will be collected from all participants at enrollment and each quarterly and annual visit.

Trial Locations

Locations (10)

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

UC Davis; 🇺🇸 Sacramento, California, United States

California Pacific Medical Center; 🇺🇸 San Francisco, California, United States

Schiff Center for Liver Diseases / University of Miami; 🇺🇸 Miami, Florida, United States

Indiana University; 🇺🇸 Indianapolis, Indiana, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Virginia Commonwealth University; 🇺🇸 Richmond, Virginia, United States

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

University Health Network; 🇨🇦 Toronto, Ontario, Canada

University Medical Center Hamburg-Eppendorf; 🇩🇪 Hamburg, Germany