Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

• Status: Completed
• Trial end date: 1999-04-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Collaborator: Mayo Clinic
• Treatments: Budesonide

Criteria

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