Primary Sclerosing Cholangitis in Children
Last updated on July 2021Recruitment
- Recruitment Status
- Not yet recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Cholangitis, Sclerosing
- Liver Diseases
- Primary Sclerosing Cholangitis
- Type
- Observational
- Design
- Observational Model: CohortTime Perspective: Prospective
Participation Requirements
- Age
- Between 2 years and 25 years
- Gender
- Both males and females
Description
Pediatric primary sclerosing cholangitis (PSC) is a rare autoimmune biliary fibrosing disease that leads to significant morbidity, the need for liver transplantation in ~50% of patients, and an increased risk for biliary and colorectal cancers in adulthood. The progression of the biliary disease in ...
Pediatric primary sclerosing cholangitis (PSC) is a rare autoimmune biliary fibrosing disease that leads to significant morbidity, the need for liver transplantation in ~50% of patients, and an increased risk for biliary and colorectal cancers in adulthood. The progression of the biliary disease in children is variable and risk factors associated with a more rapid progression of disease have not been adequately studied. Importantly, pediatric hepatologists have never previously collaborated with inflammatory bowel disease (IBD) specialists to rigorously explore interactions between colonic inflammation and liver disease. New non-invasive imaging modalities to measure fibrosis have not been explored in pediatric PSC. Furthermore, the impact that PSC has on the global functioning of children is not well understood, and likely underappreciated. The natural history of pediatric PSC is poorly understood. This study aims to determine risk factors, including activity of co-existent IBD, associated with more rapid progression of disease, characterize the impact of PSC on global functioning, define the spectrum and prognostic value of biliary tract disease and liver fibrosis based on novel imaging techniques, and establish a biobank of specimens for future mechanistic studies aimed at discovering biomarkers pertaining to etiology and severity of PSC and novel mechanisms of immunopathogenesis of disease. This comprehensive observational and longitudinal study will delineate unique aspects of the natural history and severity of pediatric PSC and of associated IBD and provide necessary data for future therapeutic trials. It aims to provide a platform to discover and validate circulating and imaging biomarkers, which may serve as surrogate endpoints in future interventional studies.
Tracking Information
- NCT #
- NCT04181138
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Investigators
- Study Chair: Cara Mack, MD Children's Hospital Colorado Study Director: Ed Doo, MD National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Study Director: Averell Sherker, MD National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Principal Investigator: John Magee, MD University of Michigan Principal Investigator: Robert Merion, MD Arbor Research Collaborative for Health