Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

Study start date: May 1, 1997

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Pathologically confirmed primary sclerosing cholangitis (PSC) meeting the following criteria: Chronic cholestatic disease for at least 6 months Liver biopsy within the past 6 months compatible with the diagnosis of PSC Intra and/or extrahepatic biliary duct obstruction, beading, or narrowing OR Pathologically confirmed primary biliary cirrhosis (PBC) that is experiencing suboptimal response to ursodeoxycholic acid and meeting the following criteria: Chronic cholestatic liver disease for at least 6 months Positive antimitochondrial antibody No biliary obstruction by ultrasound, CT, or cholangiography Prior liver biopsy compatible with diagnosis of PBC Received ursodeoxycholic acid for at least 6 months --Prior/Concurrent Therapy-- Biologic therapy: At least 3 months since prior D-penicillamine No planned transplantation for at least 1 year Chemotherapy: At least 3 months (6 months for PBC) since prior cyclosporin, colchicine, azathioprine, or methotrexate At least 6 months since prior chlorambucil (PBC only) Endocrine therapy: At least 3 months (6 months for PBC) since prior corticosteroids Surgery: No prior intraductal stones or operations on the biliary tree except cholecystectomy (PSC only) Other: At least 3 months since prior pentoxifylline, ursodeoxycholic acid, or nicotine (PSC only) At least 6 months since prior chenodeoxycholic acid (PBC only) --Patient Characteristics-- Life expectancy: At least 3 years Hematopoietic: Not specified Hepatic: Alkaline phosphatase at least 2 times upper limits of normal No chronic hepatitis B infection No hepatitis C infection No autoimmune hepatitis Bilirubin no greater than 4 mg/dL (PBC only) Cardiovascular: No severe cardiopulmonary disease Other: No concurrent advanced malignancy At least 3 months since prior inflammatory bowel disease requiring specific treatment except maintenance therapy (PSC only) No anticipated need for transplantation within 1 year Not pregnant No liver disease of other etiology such as: Chronic alcoholic liver disease Hemochromatosis Wilson's disease Congenital biliary disease Cholangiocarcinoma No recurrent ascending cholangitis requiring hospitalization more than 2 times per year (PSC only) At least 1 year since prior active peptic ulcer No recurrent variceal bleeds No spontaneous encephalopathy No diuretic-resistant ascites