Primary Sclerosing Cholangitis Primary Sclerosing Cholangitis
Conditions
Interventions
None listed
Sponsors
PSC Partners Seeking a Cure
Eligibility
Age
18 Years to 99 Years
Inclusion 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
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 antitrypsin 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 enroll2. 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 days6. UDCA dose >28 mg/kg7. 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- Patients with esophageal varices with no history or current bleeding may be enrolled providing there is no other evidence of hepatic decompensation? 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)8. Prior liver transplantation9. 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 cholangiocarcinoma or other hepatobiliary malignancy based on imaging, screening laboratory values, and/or clinical symptoms.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 48 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.15. Absence of data in medical records to assess inclusion and exclusion criteria.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| Develop a large clinical and biomarker data set to identify individual and/or composite surrogate endpoints likely to predict clinical benefit for use in the design of interventional studies in PSC. Evaluate patient reported symptoms, quality of life (QoL), and other direct patient experiences with standardized tools to determine changes over time, the association with clinical events, biomarkers, and disease progression, and confirm the psychometric properties of the PRO measures. | — |
Countries
Netherlands, United States
Contacts
Public ContactA.J.P. van der Meer
Erasmus MC, Universitair Medisch Centrum Rotterdam
Outcome results
None listed