NCT04181138 Primary Sclerosing Cholangitis in Children
| NCT ID | NCT04181138 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Arbor Research Collaborative for Health |
| Condition | Primary Sclerosing Cholangitis |
| Study Type | OBSERVATIONAL |
| Enrollment | 1,000 participants |
| Start Date | 2021-12-30 |
| Primary Completion | 2029-05-31 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 1,000 participants in total. It began in 2021-12-30 with a primary completion date of 2029-05-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
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 -
Contact & Investigator
Cara Mack, MD
STUDY CHAIR
Medical College of Wisconsin-Milwaukee
Frequently Asked Questions
Who can join the NCT04181138 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 25 Years, studying Primary Sclerosing Cholangitis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04181138 currently recruiting?
Yes, NCT04181138 is actively recruiting participants. Contact the research team at melissa.sexton@arborresearch.org for enrollment information.
Where is the NCT04181138 trial being conducted?
This trial is being conducted at Los Angeles, United States, Aurora, United States, Atlanta, United States, Chicago, United States and 8 additional locations.
Who is sponsoring the NCT04181138 clinical trial?
NCT04181138 is sponsored by Arbor Research Collaborative for Health. The principal investigator is Cara Mack, MD at Medical College of Wisconsin-Milwaukee. The trial plans to enroll 1,000 participants.