Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis - Full Text View

Sponsors and Collaborators:	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Mayo Clinic
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004842

Purpose

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.

Condition	Intervention	Phase
Cholangitis, Sclerosing Liver Cirrhosis, Biliary	Drug: budesonide	Phase I

MedlinePlus related topics: Cirrhosis

Drug Information available for: Budesonide

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Safety/Efficacy Study

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	50
Study Start Date:	October 1998

Detailed 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.

Eligibility

Ages Eligible for Study:	18 Years to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004842

Sponsors and Collaborators

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Mayo Clinic

Investigators

Study Chair:

Keith D. Lindor

Mayo Clinic

More Information

Study ID Numbers:	199/13922, MAYOC-41296, MAYOC-DK52344
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004842
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

cirrhosis
gastrointestinal disorders
primary biliary cirrhosis
primary sclerosing cholangitis
rare disease

Study placed in the following topic categories:

Biliary cirrhosis
Primary sclerosing cholangitis
Liver Diseases
Cholangitis, Sclerosing
Gastrointestinal Diseases
Fibrosis
Cholestasis
Budesonide
Rare Diseases

Liver Cirrhosis
Cholangitis
Digestive System Diseases
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Primary biliary cirrhosis
Liver Cirrhosis, Biliary

Additional relevant MeSH terms:

Anti-Inflammatory Agents
Respiratory System Agents
Physiological Effects of Drugs
Hormones, Hormone Substitutes, and Hormone Antagonists
Anti-Asthmatic Agents
Glucocorticoids
Hormones

Pharmacologic Actions
Pathologic Processes
Autonomic Agents
Therapeutic Uses
Peripheral Nervous System Agents
Bronchodilator Agents

ClinicalTrials.gov processed this record on January 16, 2009