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Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified December 1998 by Office of Rare Diseases (ORD).
Recruitment status was:  Active, not recruiting
Mayo Clinic
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: December 1998

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 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

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

Estimated Enrollment: 50
Study Start Date: October 1998
Detailed Description:


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.


Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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


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


  • 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)


  • 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


  • 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


  • 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


Wilson's disease

Congenital biliary disease


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
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Please refer to this study by its identifier: NCT00004842

Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Mayo Clinic
Study Chair: Keith D. Lindor Mayo Clinic
  More Information Identifier: NCT00004842     History of Changes
Other Study ID Numbers: 199/13922
Study First Received: February 24, 2000
Last Updated: June 23, 2005

Keywords provided by Office of Rare Diseases (ORD):
gastrointestinal disorders
primary biliary cirrhosis
primary sclerosing cholangitis
rare disease

Additional relevant MeSH terms:
Liver Cirrhosis
Cholangitis, Sclerosing
Liver Cirrhosis, Biliary
Pathologic Processes
Liver Diseases
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases
Cholestasis, Intrahepatic
Anti-Inflammatory Agents
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Hormones, Hormone Substitutes, and Hormone Antagonists processed this record on May 23, 2017