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Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

Primary Purpose

Cholangitis, Sclerosing, Liver Cirrhosis, Biliary

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
budesonide
Sponsored by
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cholangitis, Sclerosing focused on measuring cirrhosis, gastrointestinal disorders, primary biliary cirrhosis, primary sclerosing cholangitis, rare disease

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Mayo Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Budesonide

Arm Description

Oral budesonide

Outcomes

Primary Outcome Measures

Change in serum alkaline phosphatase

Secondary Outcome Measures

Full Information

First Posted
February 24, 2000
Last Updated
May 30, 2017
Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborators
Mayo Clinic
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1. Study Identification

Unique Protocol Identification Number
NCT00004842
Brief Title
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

2. Study Status

Record Verification Date
May 2017
Overall Recruitment Status
Completed
Study Start Date
May 1997 (Actual)
Primary Completion Date
April 1999 (Actual)
Study Completion Date
April 1999 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborators
Mayo Clinic

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cholangitis, Sclerosing, Liver Cirrhosis, Biliary
Keywords
cirrhosis, gastrointestinal disorders, primary biliary cirrhosis, primary sclerosing cholangitis, rare disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
22 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Budesonide
Arm Type
Experimental
Arm Description
Oral budesonide
Intervention Type
Drug
Intervention Name(s)
budesonide
Intervention Description
Oral budesonide, 9 mg daily was administered for 1 year
Primary Outcome Measure Information:
Title
Change in serum alkaline phosphatase
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Keith D. Lindor
Organizational Affiliation
Mayo Clinic
Official's Role
Study Chair
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
10655252
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.
Results Reference
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Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

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