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

NCT06297993 | Recruiting

• 1 other identifier: WIND-PSC-01
• Study Type: observational
• Target: 2,000
• Locations: 5 countries
• Sites: 16

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.

Conditions

PSC

Outcome Measures

Primary Outcomes (1)

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

  Collection of liver-related data including PSC symptoms, medical history, adverse events, and outcomes at enrollment and each quarterly and annual visit.

  Quarterly for five years from enrollment

Secondary Outcomes (3)

• Develop a large clinical and biomarker data set to identify individual and/or composite surrogate end-points likely to predict clinical benefit for use in the design of interventional studies in PSC.

  Quarterly for five years from enrollment

• Develop a large clinical and biomarker data set to identify individual and/or composite surrogate end-points likely to predict clinical benefit for use in the design of interventional studies in PSC.

  Quarterly for five years from enrollment

• Evaluate direct participant experiences with standardized tools to determine changes over time, the association with clinical events, biomarkers, and disease progression

  Quarterly for five years from enrollment

Study Arms (1)

Adult patients with a confirmed diagnosis of large duct PSC.

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

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

2000 adult participants with confirmed diagnosis of large duct PSC

You may qualify if:

• 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).
• Confirmed clinical diagnosis of large duct PSC.

You may not qualify if:

• 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
• Small-Duct PSC.
• Clinically diagnosed secondary or IgG4-related sclerosing cholangitis.
• 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
• Hospitalization in the past 7 days
• UDCA dose \>28 mg/kg
• 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

Sponsors & Collaborators

• PSC Partners Seeking a Cure: lead

Study Sites (16)

UC Davis

Sacramento, California, 95817, United States

RECRUITING

California Pacific Medical Center

San Francisco, California, 94109, United States

RECRUITING

University of California, San Francisco

San Francisco, California, 94143, United States

RECRUITING

Yale University

New Haven, Connecticut, 06510, United States

RECRUITING

Schiff Center for Liver Diseases / University of Miami

Miami, Florida, 33136, United States

RECRUITING

Indiana University

Indianapolis, Indiana, 46202, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55901, United States

RECRUITING

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

RECRUITING

University of Alberta

Edmonton, Alberta, T6G 2X8, Canada

RECRUITING

University Health Network

Toronto, Ontario, M5G2C4, Canada

RECRUITING

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

Montreal, Quebec, H2X 0A9, Canada

RECRUITING

University Medical Center Hamburg-Eppendorf

Hamburg, Hamburg, 20246, Germany

RECRUITING

University of Milano Bicocca

Milan, Monza (MB), 20900, Italy

RECRUITING

Auckland University

Auckland, New Zealand

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

A standardized procedure for collection and processing of human blood will be applied to all blood samples collected as part of the study. Barcoded samples will be stored at the clinical centers, and corresponding data will be entered into the study database. Any protocol deviations should also be recorded by each center. 30mL of blood is collected at baseline and at each annual visit.

Study Officials

• Cynthia Levy, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

• Stephen Rossi, PharmD

  PSC Partners Seeking a Cure

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Stephen Rossi, PharmD

CONTACT

3037715227; stephen@pscpartners.org

Priya Kannusamy

CONTACT

3037715227; priya@pscpartners.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 29, 2024
• First Posted: March 7, 2024
• Study Start: May 6, 2024
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): January 1, 2031
• Last Updated: February 19, 2026

Record last verified: 2026-02

Locations

CA (3); US (10); NZ (1); DE (1); IT (1)