Safety and Efficacy Study of Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis
Status:
Completed
Trial end date:
2017-06-01
Target enrollment:
Participant gender:
Summary
Primary sclerosing cholangitis (PSC), although uncommon, is a devastating and insidiously
progressive liver disease, resulting from advancing inflammation, fibrosis and obliteration
of the bile ducts in the liver, leading to cirrhosis and end-stage liver disease. Although
prognosis in children may be somewhat better than that of adults, approximately one third of
pediatric patients require transplantation by adulthood. Other than transplantation, there is
to date no therapy conclusively proven to improve the long-term outcome. Ursodeoxycholic acid
(UDCA) improves biochemical markers of liver disease, although in high doses does not clearly
improve the long-term outcome in adults, and in a recent study may have actually worsened
outcome. Childhood PSC is different from that of adult PSC in many ways, and children may
derive more short-term, as well as long-term, benefit than adults. This unique multicenter
study will carefully monitor the effects of withdrawal and restarting UDCA on liver injury
and inflammation in children with PSC. The preliminary data will help in the design of a more
definitive larger study to determine if UDCA has a beneficial role in the treatment of PSC in
children. Funding Source - FDA OOPD
Phase:
Phase 1
Details
Lead Sponsor:
University of Tennessee
Collaborators:
Ann & Robert H Lurie Children's Hospital of Chicago Baylor College of Medicine Children's Healthcare of Atlanta Children's Hospital Los Angeles Children's Hospital of Philadelphia Icahn School of Medicine at Mount Sinai Phoenix Children's Hospital Texas Children's Hospital University of California, San Francisco University of Colorado, Denver University of Pittsburgh Yale University