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Ursodiol-Methotrexate for Primary Biliary Cirrhosis
This study has been completed.
First Received: August 8, 2000   Last Updated: June 23, 2005   History of Changes
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier: NCT00006168
  Purpose

The major thrust is to determine whether treatment of patients with Primary Biliary Cirrhosis (PBC) with Ursodiol (Ursodeoxycholic Acid-UDCA) plus methotrexate (MTX) is more effective than treatment with UDCA alone.


Condition Intervention Phase
Liver Cirrhosis, Biliary
Drug: Methotrexate
Phase III

Study Type: Interventional
Study Design: Treatment, Randomized, Double-Blind, Safety/Efficacy Study

Resource links provided by NLM:


Further study details as provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):

Study Start Date: January 1994
Estimated Study Completion Date: March 2004
Detailed Description:

PBC is a chronic cholestatic liver disease, predominantly of women, in which interlobular and septal bile ducts undergo inflammation and destruction. Once initiated, the disease persists and progresses at varying rates. Neither the initiating nor perpetuating mechanisms are well understood. Current concepts of pathogenesis include (1) destruction of bile ducts is maintained and perhaps initiated by autoimmune mechanisms; (2) hydrophobic bile acids which accumulate in serum and liver cause functional and cytotoxic liver injury; (3) cytokines and lymphokines released at sites of inflammation may contribute to cell damage and fibrosis. A considerable body of evidence indicates that UDCA when fed orally leads to improvement in liver tests, in pruritus and in liver histology. There exist differences in opinion as to whether development of complications of liver disease, liver transplantation or transplant-free survival is affected. UDCA, a relatively non-toxic bile acid, when administered orally, alters the composition of the bile acid pool in factor of its enrichment with UDCA and appears to protect against the cytotoxic effects of endogenous bile acids that accumulate as a result of bile acid destruction. MTX is being shown to improve liver tests, symptoms and liver histology in a small number of precirrhotic patients with PBC. The mechanism of action is unknown but felt to be related to anti-inflammatory immunosuppressive effects of MTX. The current trial explores whether MTX improves the therapeutic effects of UDCA in PBC. Patients with PBC whose serum bilirubin is less than 3 mg%, who have been on UDCA for at least 6 months, and who satisfy a series of inclusion and exclusion criteria are stratified into 2 groups on the basis of liver histologic stage (Ludwig classification), i.e., early (Stages I and II) versus late (Stages III or IV). They are then randomized to receive either methotrexate or its placebo as a second drug while continuing to receive UDCA. The relative value of the two treatment arms is assessed by comparing their effects on symptoms, results of laboratory tests, development of complications of liver disease, histologic changes in liver, liver transplantation, and on transplant-free survival. The safety of each therapeutic regimen is also being determined.

  Eligibility

Ages Eligible for Study:   20 Years to 69 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Chronic cholestatic liver disease of at least 6 months' duration.
  • Serum alkaline phosphatase levels at least 1.5 times the upper limit of normal prior to treatment with UDCA.
  • Serum bilirubin less than 3.0 mg% prior to treatment with UDCA.
  • Serum albumin of 3.0 gram% or greater prior to treatment with UDCA.
  • Positive antimitochondrial antibody test
  • Liver biopsy within the previous 6 months after at least 6 months on UDCA (available for review, and at least 2 cm long if cirrhosis not detected) compatible with the diagnosis of PBC.
  • Ultrasound, computed tomography (CT) or cholangiography of the biliary tree which excludes biliary obstruction.

Exclusion Criteria:

  • Treatment with immunosuppressive agents including azathioprine, chlorambucil, colchicine, corticosteroids, or d-penicillamine in the preceding 3 months; or with cyclosporine, FK-506 or methotrexate in the preceding 6 months.
  • Treatment with rifampin in the preceding 3 months.
  • Serum bilirubin of 3.0 mg% or greater.
  • Serum albumin less than 3.0 gm%.
  • WBC 2,500 mm3; granulocytes 1,500 mm3; platelets 80,000mm3.
  • Ascites, hepatic encephalopathy, variceal bleed.
  • Findings by clinical, serologic and histologic evidence of liver disease of other etiology (such as chronic hepatitis B or C, autoimmune chronic active hepatitis, alcoholic liver disease, sclerosing cholangitis, drug-induced liver disease, symptomatic or obstructive gallstones).
  • Pregnancy, or if not pregnant and in the reproductive period, unwillingness to utilize an adequate form of birth control.
  • Age less than 20 or greater than 69 years.
  • Epilepsy requiring use of dilantin.
  • Malignant disease within the past 5 years (except skin cancer)
  • Anti-HIV positive. Major illnesses that could limit life span.
  • History of alcoholism during the previous 2 years.
  • Creatinine clearance less than 60 ml per minute.
  • Severe lung disease, defined as a diffusion capacity or vital capacity of less than 50 percent of predicted.
  • Patients who are both asymptomatic and have Stage I histology on liver biopsy (Ludwig classification).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00006168

Locations
United States, California
U California Medical Center
San Francisco, California, United States, 94143
Keck School of Medicine at U.S.C.
Los Angeles, California, United States, 90033
United States, Connecticut
Yale University School of Medicine
New Haven, Connecticut, United States, 06520-8019
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
United States, Missouri
Saint Louis University
St. Louis, Missouri, United States, 63104
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198-3285
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Oregon
Oregon Health Sciences University
Portland, Oregon, United States, 97201
United States, Pennsylvania
Albert Einstein Medical Center
Philadelphia, Pennsylvania, United States, 19141
United States, Texas
UT Southwestern Medical Center at Dallas
Dallas, Texas, United States, 75235-9151
United States, Virginia
Medical College of Virginia
Richmond, Virginia, United States, 23298-0711
United States, Washington
University of Washington Medical Center
Seattle, Washington, United States, 98195
Sponsors and Collaborators
  More Information

Publications:
Poupon RE, Balkau B, Eschwege E, Poupon R. A multicenter, controlled trial of ursodiol for the treatment of primary biliary cirrhosis. UDCA-PBC Study Group. N Engl J Med. 1991 May 30;324(22):1548-54.
Heathcote EJ, Cauch-Dudek K, Walker V, Bailey RJ, Blendis LM, Ghent CN, Michieletti P, Minuk GY, Pappas SC, Scully LJ, et al. The Canadian Multicenter Double-blind Randomized Controlled Trial of ursodeoxycholic acid in primary biliary cirrhosis. Hepatology. 1994 May;19(5):1149-56.
Heathcote EJ, Cauch-Dudek K, Walker V, Bailey RJ, Blendis LM, Ghent CN, Michieletti P, Minuk GY, Pappas SC, Scully LJ, et al. The Canadian Multicenter Double-blind Randomized Controlled Trial of ursodeoxycholic acid in primary biliary cirrhosis. Hepatology. 1994 May;19(5):1149-56.
Lindor KD, Dickson ER, Baldus WP, Jorgensen RA, Ludwig J, Murtaugh PA, Harrison JM, Wiesner RH, Anderson ML, Lange SM, et al. Ursodeoxycholic acid in the treatment of primary biliary cirrhosis. Gastroenterology. 1994 May;106(5):1284-90.
Combes B, Carithers RL Jr, Maddrey WC, Lin D, McDonald MF, Wheeler DE, Eigenbrodt EH, Munoz SJ, Rubin R, Garcia-Tsao G, et al. A randomized, double-blind, placebo-controlled trial of ursodeoxycholic acid in primary biliary cirrhosis. Hepatology. 1995 Sep;22(3):759-66.
Kaplan MM, Knox TA, Arora SA. Primary biliary cirrhosis treated with low-dose oral pulse methotrexate. Ann Intern Med. 1988 Sep 1;109(5):429-31. No abstract available.
Kaplan MM. Methotrexate treatment of chronic cholestatic liver diseases: friend or foe? Q J Med. 1989 Aug;72(268):757-61. No abstract available.
Kaplan MM, Knox TA. Treatment of primary biliary cirrhosis with low-dose weekly methotrexate. Gastroenterology. 1991 Nov;101(5):1332-8.
Bergasa NV, Jones A, Kleiner DE, Rabin L, Park Y, Wells MC, Hoofnagle JH. Pilot study of low dose oral methotrexate treatment for primary biliary cirrhosis. Am J Gastroenterol. 1996 Feb;91(2):295-9.
Buscher HP, Zietzschmann Y, Gerok W. Positive responses to methotrexate and ursodeoxycholic acid in patients with primary biliary cirrhosis responding insufficiently to ursodeoxycholic acid alone. J Hepatol. 1993 Apr;18(1):9-14.

Study ID Numbers: PUMPS, 5 R01 DK46602
Study First Received: August 8, 2000
Last Updated: June 23, 2005
ClinicalTrials.gov Identifier: NCT00006168     History of Changes
Health Authority: United States: Federal Government

Keywords provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
primary biliary cirrhosis
Ursodeoxycholic acid
UDCA
MTX
Hepatology
methotrexate
liver

Additional relevant MeSH terms:
Antimetabolites
Liver Diseases
Antimetabolites, Antineoplastic
Immunologic Factors
Molecular Mechanisms of Pharmacological Action
Cholestasis
Fibrosis
Antineoplastic Agents
Physiological Effects of Drugs
Liver Cirrhosis
Reproductive Control Agents
Pathologic Processes
Cholestasis, Intrahepatic
Biliary Tract Diseases
Therapeutic Uses
Abortifacient Agents
Methotrexate
Liver Cirrhosis, Biliary
Dermatologic Agents
Nucleic Acid Synthesis Inhibitors
Enzyme Inhibitors
Abortifacient Agents, Nonsteroidal
Folic Acid Antagonists
Immunosuppressive Agents
Pharmacologic Actions
Digestive System Diseases
Bile Duct Diseases
Antirheumatic Agents

ClinicalTrials.gov processed this record on November 25, 2009