Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

Conditions
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Cholangitis, Sclerosing, Liver Cirrhosis, Biliary

Intervention / Treatment
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* Drug: budesonide


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Budesonide<br>Oral budesonide | Drug: budesonide<br>* Oral budesonide, 9 mg daily was administered for 1 year<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in serum alkaline phosphatase |  | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cirrhosis, gastrointestinal disorders, primary biliary cirrhosis, primary sclerosing cholangitis, rare disease