Background

This clinical trial is designed to uncover the biological basis of primary sclerosing cholangitis (PSC), a rare liver disease. Currently, PSC has limited therapeutic options and can progress to severe complications such as cholangitis, cirrhosis, and cancer. Understanding the fundamental mechanisms of the disease is essential for developing effective therapies, making this study necessary. The research team will collect a variety of biospecimens—including blood, saliva, urine, and stool—to obtain multidimensional data.

Study Design

Participants will undergo regular clinical assessments over 12 months, including colonoscopy, upper endoscopy, and liver biopsy. Imaging modalities such as MRI, spectroscopy, and ultrasound will also be employed to comprehensively monitor disease progression. This multimodal approach is essential for precisely characterizing the pathological features of PSC and enables the identification of novel biomarkers through integrated data analysis.

Target Patient Population

The study will enroll patients aged 18 years or older with a confirmed diagnosis of PSC, including those who have not responded to standard therapy or have progressive disease. Participants will provide a three‑day dietary diary and undergo nutritional assessments to explore associations between lifestyle factors and disease. The cohort is intentionally broad to facilitate a comprehensive understanding of the diverse clinical phenotypes.

Expected Impact

If successful, the study will deliver new insights into the pathogenesis of PSC and define targetable mechanisms for next‑generation therapeutic development. The identified biomarkers are likely to be valuable for patient selection and response prediction in clinical trial settings. This scientific foundation will serve as a critical data point for pharmaceutical and biotech companies making investment decisions.