Understanding Primary Sclerosing Cholangitis in Children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study focuses on pediatric primary sclerosing cholangitis (PSC), a rare liver disease that affects bile ducts. It aims to collect medical data and specimens from children diagnosed with PSC to better understand the disease's progression and its impact on daily life. The study will explore risk factors for rapid disease progression, the relationship between PSC and inflammatory bowel disease, and utilize novel imaging techniques to assess liver fibrosis. Additionally, a biobank will be established for future research into biomarkers and disease mechanisms.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Patients with the clinical diagnosis of large or small duct PSC made at any time prior to enrollment are screened for eligibility to participate in this prospective cohort study. The site PI will determine eligibility following review of MRCP or ERCP images with the site radiologist to confirm presence of an abnormal cholangiogram at the time of diagnosis of large duct PSC. Liver histopathology obtained at the time of diagnosis of small duct PSC will be reviewed with the site pathologist prior to enrollment.

Individuals must meet all of the Inclusion criteria in order to be eligible to participate in the study:

1. Aged 2 through 25 years at time of screening.
2. Diagnosis of large duct PSC based on review of cholangiogram by MRC, ERC, or intraoperative cholangiogram (IOC) by the site radiologist and interpreted to be consistent with PSC, based on one or more of the following:

   * 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

   AND/OR
3. Diagnosis of small duct PSC based on review of liver histopathology by the site pathologist and interpreted to be compatible with PSC:

   * 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

Participants for the imaging study are eligible if they are:

1. Aged 8 through 25 years at the time of screening
2. No absolute contraindication to MRI
3. No skin condition that could be aggravated by MREL
4. Meet all other eligibility criteria of the PSC Observational Study
5. For whom none of the exclusion criteria apply

Exclusion Criteria:

An individual who meets any of the following criteria at baseline will be excluded from participation in this study.

1. History of liver transplantation
2. History bone marrow transplantation
3. History of primary or acquired immunodeficiency predisposing to secondary sclerosing cholangitis, for instance: hyper-IgM syndrome, severe combined immunodeficiency (SCID) syndrome, common variable immunodeficiency (CVID) syndrome, cartilage hair hypoplasia syndrome, or HIV/AIDS
4. History of histiocytosis, including Langerhans cell histiocytosis (LCH), or hemophagocytic lymphohistiocytosis (HLH)
5. History of ischemic cholangitis
6. History of portal vein thrombosis with biliopathy, veno-occlusive disease, or abdominal radiation vasculopathy
7. History of recurrent pyogenic cholangitis
8. History of biliary tract surgery for cholecystolithiasis prior to cholangiogram/liver biopsy evaluated to determine enrollment
9. History of biliary tract surgery for choledochal cyst
10. History of hepatocellular carcinoma, or hepatoblastoma
11. History of surgical biliary trauma
12. History of congenital cytomegalovirus (CMV) hepatitis
13. History of Sickle Cell Disease
14. History of cystic fibrosis, biliary atresia, Caroli disease/congenital hepatic fibrosis, or progressive familial intrahepatic cholestasis type 3/MDR3 disease
15. History of cardiac hepatopathy.
16. History of metabolic disorders, including Wilson's disease, glycogen storage disorder, Alpha-1 Antitrypsin deficiency
17. Diagnosis of systemic lupus erythematosus (SLE)
18. Concurrent pregnancy at the time of enrollment -

Where it's running

• Children's Hospital of Los Angeles
  Los Angeles, California, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• Children's Healthcare of Atlanta
  Atlanta, Georgia, United States Recruiting
• Ann & Robert H Lurie Children's Hospital
  Chicago, Illinois, United States Recruiting
• Cincinnati Children's Hospital Medical
  Cincinnati, Ohio, United States Recruiting
• The Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Principal investigator: John Magee, MD — University of Michigan
• Study coordinator: Melissa Sexton, BBA, CCRP
• Email: melissa.sexton@arborresearch.org
• Phone: 734-693--3811

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.