Observational Study of Primary Sclerosing Cholangitis in Children

This study is looking to learn more about Primary Sclerosing Cholangitis (PSC) in children, a rare liver disease that damages the liver's bile ducts (tiny tubes that carry bile from the liver to the small intestine). Researchers want to understand how PSC progresses and identify factors that might make the disease get worse faster. They will collect medical information and other data from children and young adults with PSC. The goal is to better understand the different types of PSC, including those with or without Inflammatory Bowel Disease (IBD) or Autoimmune Hepatitis (AIH). This is an observational study, meaning no new treatments are being tested. You may be able to join if you are between 2 and 25 years old and have a diagnosis of PSC. The study aims to enroll 1000 participants, but its current status is unclear.

Study design
This is an observational study aiming to enroll 1000 participants. It will characterize major phenotypes of PSC.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be characterized for up to 10 years.

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NCT04181138

Primary Sclerosing Cholangitis in Children

Recruiting
Not specifiedAges 2–25Observational
Arbor Research Collaborative for Health
~1,000 participants
Updated 2026-07-14 on ClinicalTrials.gov

At a glance

Recruiting sites
9 of 12 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To characterize the major phenotypes of PSC including patients with large duct or small duct disease, with and without Inflammatory Bowel Disease (IBD), and with and without Auto Immune Hepatitis (AIH)
Measured over up to 10 years
Primary Sclerosing Cholangitis
Liver Diseases
Cholangitis, Sclerosing
12 sites across 11 states
Pennsylvania2
California1
Colorado1
Georgia1
Illinois1
Indiana1
Ohio1
Texas1
  • Cara Mack, MD · STUDY_CHAIR · Medical College of Wisconsin-Milwaukee
  • Ed Doo, MD · STUDY_DIRECTOR · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Katrina Loh, MD · STUDY_DIRECTOR · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • John Magee, MD · PRINCIPAL_INVESTIGATOR · University of Michigan
  • Lisa Henn, PhD · PRINCIPAL_INVESTIGATOR · Arbor Research Collaborative for Health

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Eligibility criteria

Inclusion

Focal structuring of the bile duct(s)
Dominant stricture of the common bile duct
Saccular dilatation of bile duct(s)
Beaded appearance of bile duct(s)
Pruning appearance of the distal bile duct branches
Probable small duct PSC: biopsy with ≥3 of 5 criteria: periductal edema, concentric inflammation, bile duct injury, ductular reaction, and neutrophils in bile ducts (cholangitis) OR...
Definitive small duct PSC: Periductal fibrosis/ "onion skinning" around interlobular bile ducts or smaller profiles 4. Stated willingness to comply with all study procedures and availability for the duration of the study. 5. Able to provide informed consent/assent
  • To characterize the major phenotypes of PSC including patients with large duct or small duct disease, with and without Inflammatory Bowel Disease (IBD), and with and without Auto Immune Hepatitis (AIH)up to 10 years

    Data will be collected on all phenotypes of PSC but attention is focused on how the intestinal inflammation and clinical activity of Inflammatory Bowel Disease (IBD) affect the progression of PSC, better classification of patients with features of Auto Immune Hepatitis (AIH), and the implications of bacterial cholangitis amongst all PSC phenotypes. Collection of retrospective clinical and laboratory data from the time of diagnosis of PSC and annual timepoints thereafter. Information regarding clinically important timepoints, laboratory data and FibroScan Liver Stiffness Measurements (LSM) are collected prospectively. Slides/images (if available) from each liver biopsy obtained at the time of diagnosis of PSC and thereafter and from the explanted liver recovered at the time of liver transplantation will undergo central review. Cholangiography results (MRCP, ERCP) will also undergo central review.