RECRUITING

Primary Sclerosing Cholangitis in Children

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2021-12-30
Study Completion:2029-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04181138

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.

Ages Eligible for Study:2 Years to 25 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Aged 2 through 25 years at time of screening.
  2. 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:
  3. * Focal structuring of the bile duct(s)
  4. * Dominant stricture of the common bile duct
  5. * Saccular dilatation of bile duct(s)
  6. * Beaded appearance of bile duct(s)
  7. * Pruning appearance of the distal bile duct branches
  8. 3. Diagnosis of small duct PSC based on review of liver histopathology by the site pathologist and interpreted to be compatible with PSC:
  9. * 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...
  10. * Definitive small duct PSC: Periductal fibrosis/ "onion skinning" around interlobular bile ducts or smaller profiles
  11. 4. Stated willingness to comply with all study procedures and availability for the duration of the study.
  12. 5. Able to provide informed consent/assent
  13. 1. Aged 8 through 25 years at the time of screening
  14. 2. No absolute contraindication to MRI
  15. 3. No skin condition that could be aggravated by MREL
  16. 4. Meet all other eligibility criteria of the PSC Observational Study
  17. 5. For whom none of the exclusion criteria apply
  1. 1. History of liver transplantation
  2. 2. History bone marrow transplantation
  3. 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. 4. History of histiocytosis, including Langerhans cell histiocytosis (LCH), or hemophagocytic lymphohistiocytosis (HLH)
  5. 5. History of ischemic cholangitis
  6. 6. History of portal vein thrombosis with biliopathy, veno-occlusive disease, or abdominal radiation vasculopathy
  7. 7. History of recurrent pyogenic cholangitis
  8. 8. History of biliary tract surgery for cholecystolithiasis prior to cholangiogram/liver biopsy evaluated to determine enrollment
  9. 9. History of biliary tract surgery for choledochal cyst
  10. 10. History of hepatocellular carcinoma, or hepatoblastoma
  11. 11. History of surgical biliary trauma
  12. 12. History of congenital cytomegalovirus (CMV) hepatitis
  13. 13. History of Sickle Cell Disease
  14. 14. History of cystic fibrosis, biliary atresia, Caroli disease/congenital hepatic fibrosis, or progressive familial intrahepatic cholestasis type 3/MDR3 disease
  15. 15. History of cardiac hepatopathy.
  16. 16. History of metabolic disorders, including Wilson's disease, glycogen storage disorder, Alpha-1 Antitrypsin deficiency
  17. 17. Diagnosis of systemic lupus erythematosus (SLE)
  18. 18. Concurrent pregnancy at the time of enrollment -

Contacts and Locations

Study Contact

Terese A Howell, BS, CCRC
CONTACT
734-476-5340
terri.howell@arborresearch.org
Sayori Suda-Wilson, BS, RD
CONTACT
734-678-5070
sayori.suda-wilson@arborresearch.org

Principal Investigator

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 Locations (Sites)

Children's Hospital of Los Angeles
Los Angeles, California, 90027
United States
Children's Hospital Colorado
Aurora, Colorado, 80045
United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
United States
Ann & Robert H Lurie Children's Hospital
Chicago, Illinois, 60611
United States
Riley Hospital for Children
Indianapolis, Indiana, 46202
United States
Cincinnati Children's Hospital Medical
Cincinnati, Ohio, 45229
United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
United States
Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, 77030
United States
The University of Utah
Salt Lake City, Utah, 84113
United States
Seattle Children's Hospital
Seattle, Washington, 98105
United States

Collaborators and Investigators

Sponsor: 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

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-12-30
Study Completion Date2029-05

Study Record Updates

Study Start Date2021-12-30
Study Completion Date2029-05

Terms related to this study

Keywords Provided by Researchers

  • Pediatric Liver Disease
  • Primary Sclerosing Cholangitis

Additional Relevant MeSH Terms

  • Primary Sclerosing Cholangitis
  • Liver Diseases
  • Cholangitis, Sclerosing