Clinical trial NCT06297993: Global Prospective, Observational Cohort of Adult Patients With Primary Sclerosing Cholangitis (WIND-PSC Study)
Official title: A Global Multi-Center Prospective Observational Cohort to Support Drug Development in Adult Patients With Primary Sclerosing Cholangitis (WIND-PSC)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: PSC
Lead sponsor: PSC Partners Seeking a Cure
Sponsor class: OTHER
First posted: 2024-03-07
Enrollment: 2000
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 2000 adult participants with confirmed diagnosis of large duct PSC

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Develop interventional clinical trial external control using clinical and biomarker data.
timeFrame: Quarterly for five years from enrollment
Secondary outcomes:
measure: Identify alternative endpoints to predict clinical benefit using large clinical and biomarker data.
timeFrame: Quarterly for five years from enrollment
measure: Changes in patient reported outcomes over time as assessed by Patient Reported Outcomes surveys.
timeFrame: Quarterly for five years from enrollment
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Miami
country: United States
facility: Schiff Center for Liver Diseases / University of Miami
state: Florida
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Toronto
country: Canada
facility: University Health Network
state: Ontario
city: Hamburg
country: Germany
facility: University Medical Center Hamburg-Eppendorf
Study officials:
affiliation: University of Miami
name: Cynthia Levy, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: PSC Partners Seeking a Cure
name: Stephen Rossi, PharmD
role: PRINCIPAL_INVESTIGATOR
References: