Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

Liver Cirrhosis, Biliary Clinical Trial

Official title:

Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004842
• Other study ID #: 199/13922
• Secondary ID: MAYOC-41296R03DK
• Status: Completed
• Phase: Phase 1
• First received: February 24, 2000
• Last updated: May 30, 2017
• Start date: May 1997
• Est. completion date: April 1999

Study information

• Verified date: May 2017
• Source: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: April 1999
• Est. primary completion date: April 1999
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Cholangitis
• Cholangitis, Sclerosing
• Fibrosis
• Liver Cirrhosis
• Liver Cirrhosis, Biliary

Intervention

Drug:
• budesonide
Oral budesonide, 9 mg daily was administered for 1 year

Locations

Country	Name	City	State
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (2)

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

Country where clinical trial is conducted

United States, 

References & Publications (1)

Angulo P, Jorgensen RA, Keach JC, Dickson ER, Smith C, Lindor KD. Oral budesonide in the treatment of patients with primary biliary cirrhosis with a suboptimal response to ursodeoxycholic acid. Hepatology. 2000 Feb;31(2):318-23. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in serum alkaline phosphatase		1 year