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

PHASE1CompletedINTERVENTIONAL
Enrollment

27

Participants

Timeline

Start Date

October 31, 2010

Primary Completion Date

December 31, 2016

Study Completion Date

June 30, 2017

Conditions
Primary Sclerosing Cholangitis
Interventions
DRUG

ursodeoxycholic acid (UDCA)

Pediatric PSC patients already receiving UDCA therapy will enter a four-phase trial consisting of baseline data collection (phase I, 4 weeks), 50% reduction in UDCA dose (phase II, 4 weeks), discontinuation of UDCA (phase III, 8 weeks) and reinstitution of therapy at a dose of 20 mg/kg/day (phase IV, 8 weeks). Surveillance and endpoint evaluation for each phase will include liver chemistries and clinical data. Comparisons will be made between baseline and the end of phase III (primary outcome) and between the end of phase III and the end of phase IV (secondary outcome). Serum cytokine biomarkers will be measured and compared between baseline and the end of phase III and between the end of phases III and IV.

Trial Locations (12)

15213

University of Pittsburgh, Pittsburgh

19104

Children's Hospital of Philadelphia, Philadelphia

30322

Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta

38103

University of Tennessee Health Science Center, Memphis

60614

Northwestern University, Chicago

77030

Texas Children's Hospital, Houston

80045

Children's Hospital Colorado, Aurora

85016

Phoenix Children's Hospital, Phoenix

90027

Children's Hospital Los Angeles, Los Angeles

94143

University of California, San Francisco, San Francisco

06510

Yale New Haven Children's Hospital, New Haven

07624

Mount Sinai School of Medicine, New York

Sponsors
All Listed Sponsors
collaborator

Icahn School of Medicine at Mount Sinai

OTHER

collaborator

Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

collaborator

University of Colorado, Denver

OTHER

collaborator

University of California, San Francisco

OTHER

collaborator

University of Pittsburgh

OTHER

collaborator

Phoenix Children's Hospital

OTHER

collaborator

Children's Hospital of Philadelphia

OTHER

collaborator

Children's Healthcare of Atlanta

OTHER

collaborator

Children's Hospital Los Angeles

OTHER

collaborator

Baylor College of Medicine

OTHER

collaborator

Yale University

OTHER

lead

University of Tennessee

OTHER