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Completed Phase 1 Interventional

Safety and Efficacy Study of Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis

ClinicalTrials.gov ID: NCT01088607

Public ClinicalTrials.gov record NCT01088607. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 5, 2026, 2:22 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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

Study identification

NCT ID
NCT01088607
Recruitment status
Completed
Study type
Interventional
Phase
Phase 1
Lead sponsor
University of Tennessee
Other
Enrollment
27 participants

Conditions and interventions

Eligibility (public fields only)

Age range
5 Years to 21 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Sep 30, 2010
Primary completion
Nov 30, 2016
Completion
May 31, 2017
Last update posted
Oct 25, 2017

2010 – 2017

United States locations

U.S. sites
12
U.S. states
10
U.S. cities
12
Facility City State ZIP Site status
Phoenix Children's Hospital Phoenix Arizona 85016
Children's Hospital Los Angeles Los Angeles California 90027
University of California, San Francisco San Francisco California 94143
Children's Hospital Colorado Aurora Colorado 80045
Yale New Haven Children's Hospital New Haven Connecticut 06510
Children's Healthcare of Atlanta, Emory University School of Medicine Atlanta Georgia 30322
Northwestern University Chicago Illinois 60614
Mount Sinai School of Medicine New York New York 07624
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19104
University of Pittsburgh Pittsburgh Pennsylvania 15213
University of Tennessee Health Science Center Memphis Tennessee 38103
Texas Children's Hospital Houston Texas 77030

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01088607, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Oct 25, 2017 · Synced Sep 5, 2026

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The complete protocol, eligibility criteria, and contact information for NCT01088607 live on ClinicalTrials.gov.

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