Clinical trial NCT00004842: Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis
Official title: Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Cholangitis, Sclerosing
Liver Cirrhosis, Biliary
Keywords: cirrhosis
gastrointestinal disorders
primary biliary cirrhosis
primary sclerosing cholangitis
rare disease
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor class: NIH
Collaborators: name: Mayo Clinic
First posted: 2000-02-25
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

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 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

Primary outcomes:
measure: Change in serum alkaline phosphatase
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Keith D. Lindor
role: STUDY_CHAIR
References:
citation: 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. doi: 10.1002/hep.510310209.