Primary sclerosing cholangitis.
Gideon M Hirschfield, Tom H Karlsen, Keith D Lindor...
https://pubmed.ncbi.nlm.nih.gov/23810223Actively Recruiting
Age: 2Years - 25Years
All Genders
ID04181138
Prospective Observational Study of Primary Sclerosing Cholangitis (PSC) in Children
Led by Arbor Research Collaborative for Health · Updated on 2026-06-04
1000
Participants Needed
12
Research Sites
N/A
Total Duration
On this page
Sponsors
A
Arbor Research Collaborative for Health
Lead Sponsor
N
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Primary sclerosing cholangitis (PSC) is a rare liver disease affecting children, where damage to the bile ducts causes bile to back up and scar the liver. This research aims to better understand how PSC develops and progresses in children, including how symptoms impact daily life, and to identify factors that may cause faster disease progression. The study also seeks to explore how PSC interacts with inflammatory bowel disease (IBD) and to discover new markers that could help predict disease severity and progression. Children aged 2 to 25 years diagnosed with either large or small duct PSC are included in this long-term observational study. Medical information, imaging, and biological samples like DNA and blood will be collected to support research. The study also uses new imaging methods to measure liver fibrosis and bile duct injury. Participants who qualify for an imaging sub-study must be between 8 and 25 years old without MRI contraindications or skin conditions that could be worsened by the imaging technique. Participants will be followed for up to 10 years, during which researchers will gather data on PSC symptoms, liver and bile duct changes through imaging, and the impact of the disease on overall functioning. Biological specimens will be stored in a biobank for future research. The primary outcome is to characterize the main PSC types including those with or without IBD or autoimmune hepatitis. Secondary outcomes include symptom effects, imaging correlations, and the development of a specimen repository. This comprehensive approach aims to provide insights for future treatments and improve understanding of pediatric PSC.
CONDITIONS
Brief Title
Primary Sclerosing Cholangitis in Children
Who Can Participate
Age: 2Years - 25Years
All Genders
Eligibility Criteria
You may qualify if you...
- Participants aged 2 through 25 years at the time of screening
- Clinical diagnosis of large duct PSC confirmed by imaging reviewed with a radiologist
- Diagnosis of small duct PSC confirmed by liver biopsy reviewed with a pathologist
- Willingness to comply with all study procedures and available for study duration
- Able to provide informed consent or assent
- For imaging sub-study: aged 8 through 25 years, no contraindications to MRI, no skin conditions aggravated by imaging, and meet other PSC study eligibility criteria
You will not qualify if you...
- History of liver transplantation
- History of bone marrow transplantation
- History of primary or acquired immunodeficiency disorders such as HIV/AIDS, SCID, or CVID
- History of histiocytosis or hemophagocytic lymphohistiocytosis
- History of ischemic cholangitis
- History of portal vein thrombosis with biliopathy, veno-occlusive disease, or abdominal radiation vasculopathy
- History of recurrent pyogenic cholangitis
- History of biliary tract surgery for cholecystolithiasis or choledochal cyst
- History of hepatocellular carcinoma or hepatoblastoma
- History of surgical biliary trauma
- History of congenital cytomegalovirus hepatitis
- History of sickle cell disease
- History of cystic fibrosis, biliary atresia, Caroli disease, or progressive familial intrahepatic cholestasis type 3
- History of cardiac hepatopathy
- History of metabolic disorders including Wilson's disease, glycogen storage disorder, or Alpha-1 Antitrypsin deficiency
- Diagnosis of systemic lupus erythematosus (SLE)
- Current pregnancy at enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Long-term Monitoring
Duration - Up to 10 years
Participants with primary sclerosing cholangitis (PSC) are observed over time to understand disease progression, symptoms impact, and liver fibrosis using imaging and other assessments.
Regular visits over the study duration as scheduled by the study team
Trial Site Locations
Total: 12 locations
1
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Ann & Robert H Lurie Children's Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
5
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Completed
6
Cincinnati Children's Hospital Medical
Cincinnati, Ohio, United States, 45229
Actively Recruiting
7
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Completed
9
Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, United States, 77030
Actively Recruiting
10
The University of Utah
Salt Lake City, Utah, United States, 84113
Actively Recruiting
11
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
12
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Completed
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Research Team
M
Melissa Sexton, BBA, CCRP
M
Melissa Sexton, BBA, CCRP
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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Published Research Related To This Trial
Pathogenesis of primary sclerosing cholangitis and advances in diagnosis and management.
John E Eaton, Jayant A Talwalkar, Konstantinos N Lazaridis...
https://pubmed.ncbi.nlm.nih.gov/23827861Incidence, clinical spectrum, and outcomes of primary sclerosing cholangitis in a United States community.
Kiran Bambha, W Ray Kim, Jayant Talwalkar...
https://pubmed.ncbi.nlm.nih.gov/14598252Primary sclerosing cholangitis, autoimmune hepatitis, and overlap in Utah children: epidemiology and natural history.
Mark Deneau, M Kyle Jensen, John Holmen...
https://pubmed.ncbi.nlm.nih.gov/23686586The prevalence, incidence and natural history of primary sclerosing cholangitis in an ethnically diverse population.
Elaine Toy, Sripriya Balasubramanian, Carlo Selmi...
https://pubmed.ncbi.nlm.nih.gov/21767410Prevalence of primary sclerosing cholangitis in patients with ulcerative colitis.
R Olsson, A Danielsson, G Järnerot...
https://pubmed.ncbi.nlm.nih.gov/2013375The IBD and PSC Phenotypes of PSC-IBD.
Amanda Ricciuto, Binita M Kamath, Anne M Griffiths
https://pubmed.ncbi.nlm.nih.gov/29594739Diagnosis and Management of Pediatric Autoimmune Liver Disease: ESPGHAN Hepatology Committee Position Statement.
Giorgina Mieli-Vergani, Diego Vergani, Ulrich Baumann...
https://pubmed.ncbi.nlm.nih.gov/29356770Sclerosing cholangitis in children.
D Debray, D Pariente, E Urvoas...
https://pubmed.ncbi.nlm.nih.gov/8283375Long-term follow-up of children and adolescents with primary sclerosing cholangitis and autoimmune sclerosing cholangitis.
Vratislav Smolka, Eva Karaskova, Oksana Tkachyk...
https://pubmed.ncbi.nlm.nih.gov/27498582