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.
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.
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.
2000 adult participants with confirmed diagnosis of large duct PSC
Cohort
Other
Not listed
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.
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.
Collection of biomarkers at enrollment and each annual visit.
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.
Collection of imaging at enrollment and each annual visit.
Evaluate direct participant experiences with standardized tools to determine changes over time, the association with clinical events, biomarkers, and disease progression
Patient Reported Outcomes surveys will be collected from all participants at enrollment and each quarterly and annual visit.
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