Primary Sclerosing Cholangitis Drug Study – Phase I

Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: A Pilot Withdrawal/ Reinstitution Trial

Protocol Description

This multi-center study carefully monitors the effects of a therapy involving use of ursodeoxycholic acid (UDCA) on liver injury and inflammation in children with primary sclerosing cholangitis (PSC). Although uncommon, PSC is a devastating and insidiously progressive liver disease that results from advancing inflammation, scarring and blockage of the bile ducts, leading to cirrhosis and end-stage liver disease. In the case of children with PSC, approximately one third require liver transplantation by adulthood. Researchers hope to determine if UDCA works differently in children than it does in adults and to determine the effects that UDCA has on liver disease in children.

Study Level: Phase I

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting patients of both genders up to 21 years of age with PSC who have been on UDCA treatment for at least 6 months and have stable liver tests that are normal or near normal.
Males and Females: Up to age 21

Requirements

The study entails four phases. Initially, UDCA treatment continues for 4 weeks, with monitoring to establish baseline measurements and confirm the liver tests are normal or near normal. In subsequent phases the dose is reduced, then stopped, then resumed at a standard level. Blood test monitoring, physical examinations and careful clinical monitoring of disease activity are provided throughout the study periods.
Visits: 7
Duration: Approximately 24 weeks

Status: Open to Enrollment

Source(s) of Support

U. S. Food and Drug Administration

Primary Investigator

Robert H. Squires, MD

Contact Information

For more information about the study or enrollment, please contact:
Kathy Bukauskas, RN, CCRC
412-692-7703