Clinical trial NCT00059202: Trial of High-dose Urso in Primary Sclerosing Cholangitis
Official title: Multicentered Randomized Trial of High-dose Urso in Primary Sclerosing Cholangitis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Primary Sclerosing Cholangitis
Keywords: Urso
PSC
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor class: NIH
First posted: 2003-04-22
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a multicenter, randomized, controlled trial of high dose ursodiol versus placebo for patients with primary sclerosing cholangitis (PSC). The average duration of follow-up will be approximately five years with important clinical endpoints such as death, eligibility for liver transplantation, changes in histology and cholangiogram as well as liver biochemistries and quality of life data collected.


Eligibility criteria: * Chronic cholestatic disease of at least six months' duration.
* Serum alkaline phosphatase at least 1 ½ times the upper limits of normal.
* Retrograde, operative, percutaneous, or magnetic resonance cholangiography demonstrating intrahepatic and/or extrahepatic biliary duct obstruction, beading, or narrowing consistent with PSC within one year of the study entry.
* Liver biopsy in the previous one year which is available for review and compatible with the diagnosis of PSC. Compatible biopsy features include fibrous cholangitis, ductopenia with periportal inflammation and biliary fibrosis.

Primary outcomes:
measure: Number of Participants With Treatment Failure
timeFrame: 5 years
Secondary outcomes:
measure: Number of Deaths
timeFrame: 5 years
measure: Number of Participants Who Have a Liver Transplantation
timeFrame: 5 years
measure: Number of Participants Meeting Minimal Listing Criteria for Liver Transplantation
timeFrame: 5 years
measure: Number of Participants Who Developed Cholangiocarcinoma
timeFrame: 5 years
measure: Number of Participants Who Developed Varices
timeFrame: 5 years
measure: Number of Participants Who Developed Cirrhosis
timeFrame: 5 years
measure: Alkaline Phosphatase at 12 Months
timeFrame: 12 months
measure: Alkaline Phosphatase at 24 Months
timeFrame: 24 months
measure: Alkaline Phosphatase at 36 Months
timeFrame: 36 months
measure: Aspartate Aminotransferase at 12 Months
timeFrame: 12 months
measure: Aspartate Aminotransferase at 24 Months
timeFrame: 24 months
measure: Aspartate Aminotransferase at 36 Months
timeFrame: 36 months
measure: Bilirubin at 12 Months
timeFrame: 12 months
measure: Bilirubin at 24 Months
timeFrame: 24 months
measure: Bilirubin at 36 Months
timeFrame: 36 months
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis University
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Richmond
country: United States
facility: Medical College of Virginia
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
Study officials:
References:
citation: Lindor KD, Kowdley KV, Luketic VA, Harrison ME, McCashland T, Befeler AS, Harnois D, Jorgensen R, Petz J, Keach J, Mooney J, Sargeant C, Braaten J, Bernard T, King D, Miceli E, Schmoll J, Hoskin T, Thapa P, Enders F. High-dose ursodeoxycholic acid for the treatment of primary sclerosing cholangitis. Hepatology. 2009 Sep;50(3):808-14. doi: 10.1002/hep.23082.