Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis
This trial is active, not recruiting.
|Conditions||cholangitis, sclerosing, liver cirrhosis, biliary|
|Sponsor||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|
|Start date||October 1998|
|Trial size||50 participants|
|Trial identifier||NCT00004842, 199/13922, MAYOC-41296, MAYOC-DK52344|
OBJECTIVES: I. Assess the safety and effectiveness of budesonide in patients with primary sclerosing cholangitis or primary biliary cirrhosis experiencing a suboptimal response to ursodeoxycholic acid.
II. Estimate the efficacy of this therapy in these patient groups as a means of evaluating the feasibility of a long-term randomized trial.
|Endpoint classification||safety/efficacy study|
Male or female participants from 18 years up to 70 years old.
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
|Description||PROTOCOL OUTLINE: Patients receive budesonide by mouth 3 times daily for a minimum of 6 months. If liver biochemistries become normal, dosage is reduced to once daily. Treatment discontinues after 1 year.|
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