Primary Sclerosing Cholangitis in Children

Description

Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. In people with PSC, the bile backs up into the liver and will damage it, causing scarring of the liver. The purposes of this study are to: * Collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly. * Ask questions about how PSC symptoms affect your child's life to learn more about its impact on your child's daily functioning * Children with PSC who are seen at one of the participating clinical sites in the Childhood Liver Disease Research Network (ChiLDReN) will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC.

Conditions

Primary Sclerosing Cholangitis, Liver Diseases, Cholangitis, Sclerosing

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Study Overview

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Study Details

Study overview

Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. In people with PSC, the bile backs up into the liver and will damage it, causing scarring of the liver. The purposes of this study are to: * Collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly. * Ask questions about how PSC symptoms affect your child's life to learn more about its impact on your child's daily functioning * Children with PSC who are seen at one of the participating clinical sites in the Childhood Liver Disease Research Network (ChiLDReN) will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC.

Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children

Primary Sclerosing Cholangitis in Children

Condition
Primary Sclerosing Cholangitis
Intervention / Treatment

-

Contacts and Locations

Los Angeles

Children's Hospital of Los Angeles, Los Angeles, California, United States, 90027

Aurora

Children's Hospital Colorado, Aurora, Colorado, United States, 80045

Atlanta

Children's Healthcare of Atlanta, Atlanta, Georgia, United States, 30322

Chicago

Ann & Robert H Lurie Children's Hospital, Chicago, Illinois, United States, 60611

Indianapolis

Riley Hospital for Children, Indianapolis, Indiana, United States, 46202

Cincinnati

Cincinnati Children's Hospital Medical, Cincinnati, Ohio, United States, 45229

Philadelphia

The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Pittsburgh

UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States, 15224

Houston

Texas Children's Hospital (Baylor College of Medicine), Houston, Texas, United States, 77030

Salt Lake City

The University of Utah, Salt Lake City, Utah, United States, 84113

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 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
  • 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
  • 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
  • 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 -

Ages Eligible for Study

2 Years to 25 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Arbor Research Collaborative for Health,

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)

Averell Sherker, 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

Study Record Dates

2029-05