NCT04181138 · Arbor Research Collaborative for Health
Primary Sclerosing Cholangitis in Children
What this study is about
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.
View original scientific 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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:
- Aged 2 through 25 years at time of screening.
- 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
- 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
- Stated willingness to comply with all study procedures and availability for the duration of the study.
- Able to provide informed consent/assent Participants for the imaging study are eligible if they are:
- Aged 8 through 25 years at the time of screening
- No absolute contraindication to MRI
- No skin condition that could be aggravated by MREL
- Meet all other eligibility criteria of the PSC Observational Study
- 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.
- History of liver transplantation
- History bone marrow transplantation
- 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
- History of histiocytosis, including Langerhans cell histiocytosis (LCH), or hemophagocytic lymphohistiocytosis (HLH)
- 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 prior to cholangiogram/liver biopsy evaluated to determine enrollment
- History of biliary tract surgery for choledochal cyst
- History of hepatocellular carcinoma, or hepatoblastoma
- History of surgical biliary trauma
- History of congenital cytomegalovirus (CMV) hepatitis
- History of Sickle Cell Disease
- History of cystic fibrosis, biliary atresia, Caroli disease/congenital hepatic fibrosis, or progressive familial intrahepatic cholestasis type 3/MDR3 disease
- History of cardiac hepatopathy.
- History of metabolic disorders, including Wilson's disease, glycogen storage disorder, Alpha-1 Antitrypsin deficiency
- Diagnosis of systemic lupus erythematosus (SLE)
- Concurrent pregnancy at the time of enrollment -
Where
- Los Angeles, California
- Aurora, Colorado
- Atlanta, Georgia
- Chicago, Illinois
- Indianapolis, Indiana
- Cincinnati, Ohio
- Philadelphia, Pennsylvania
- Pittsburgh, Pennsylvania
- Houston, Texas
- Salt Lake City, Utah
- Seattle, Washington
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 4, 2026 · Source of record for eligibility and locations