Trial of High-dose Urso in Primary Sclerosing Cholangitis

Phase 3TerminatedNCT00059202

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)150 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

150

Conditions

Primary Sclerosing Cholangitis

Interventions

Ursodeoxycholic AcidDRUG

Ursodeoxycholic Acid 28-30 mg/kg/day in divided doses

PlaceboDRUG

Placebo for Urso

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

* 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.

Locations (7)

Mayo Clinic

Scottsdale, Arizona, United States

Mayo Clinic

Jacksonville, Florida, United States

Mayo Clinic

Rochester, Minnesota, United States

St. Louis University

St Louis, Missouri, United States

Outcomes

Primary Outcomes

Number of Participants With Treatment Failure

Treatment failure was defined as death, transplantation, meeting minimal listing criteria, development of varices, cholangiocarcinoma, or progression to cirrhosis

Time frame: 5 years

Secondary Outcomes

Number of Deaths

Death at any time up to 5 years

Time frame: 5 years

Number of Participants Who Have a Liver Transplantation

Liver transplantation at any time up to 5 years

Time frame: 5 years

Number of Participants Meeting Minimal Listing Criteria for Liver Transplantation

Meeting minimal listing criteria for liver transplantation at any time up to 5 years. Minimal listing criteria are defined as a MELD score of 14 or higher. The Model For End-Stage Liver Disease (MELD) is a numeric score with a range of 6 to 40, where a higher score means worse liver disease.

Time frame: 5 years

Number of Participants Who Developed Cholangiocarcinoma

Development of cholangiocarcinoma at any time up to 5 years

Time frame: 5 years

Number of Participants Who Developed Varices

Development of esophogeal and/or gastric varices

Time frame: 5 years

Number of Participants Who Developed Cirrhosis

Development of cirrhosis based on liver biopsy

Time frame: 5 years

Alkaline Phosphatase at 12 Months

Data from ClinicalTrials.gov

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