Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

NCT00004842 | Completed Phase 1 FDA Drug

• 3 other identifiers: 199/13922MAYOC-41296R03DK052344
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

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.

Conditions

Cholangitis, Sclerosing; Liver Cirrhosis, Biliary

Keywords

cirrhosis; gastrointestinal disorders; primary biliary cirrhosis; primary sclerosing cholangitis; rare disease

Outcome Measures

Primary Outcomes (1)

• Change in serum alkaline phosphatase

  1 year

Study Arms (1)

Budesonide

EXPERIMENTAL

Oral budesonide

Drug: budesonide

Interventions

budesonide DRUG

Oral budesonide, 9 mg daily was administered for 1 year

Budesonide

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): lead
• Mayo Clinic: collaborator

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related 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. doi: 10.1002/hep.510310209.

  PMID: 10655252 RESULT

MeSH Terms

Conditions

Cholangitis, Sclerosing; Liver Cirrhosis, Biliary; Fibrosis; Gastrointestinal Diseases; Rare Diseases

Interventions

Budesonide

Condition Hierarchy (Ancestors)

Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Cholestasis, Intrahepatic; Cholestasis; Liver Diseases; Liver Cirrhosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Disease Attributes

Intervention Hierarchy (Ancestors)

Pregnenediones; Pregnenes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Keith D. Lindor

  Mayo Clinic

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 24, 2000
• First Posted: February 25, 2000
• Study Start: May 1, 1997
• Primary Completion: April 1, 1999
• Study Completion: April 1, 1999
• Last Updated: May 31, 2017

Record last verified: 2017-05

Locations

US (1)