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Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis

This study has been completed.
Tufts Medical Center
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: December 2001


I. Compare the efficacy of low-dose oral pulse methotrexate (MTX) and ursodiol versus colchicine and ursodiol in patients with primary biliary cirrhosis (PBC).

II. Determine the optimum dose and duration of MTX treatment.

III. Investigate the role of fibrogenic cytokines (FC) in PBC pathogenesis and the effect of treatment on FC production.

Condition Intervention Phase
Liver Cirrhosis, Biliary
Drug: colchicine
Drug: methotrexate
Drug: ursodiol
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: Phase III Randomized, Double-Blind, Placebo-Controlled Study of Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis

Resource links provided by NLM:

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

Estimated Enrollment: 90
Study Start Date: November 1989
Detailed Description:


This is a randomized, double-blind study. Patients are stratified by prior/concurrent medical management.

Patients in the first group are treated with oral methotrexate 3 times a week and a daily oral placebo.

Patients in the second group are treated with daily oral colchicine and an oral placebo 3 times a week.

Therapy continues for 10 years. Beginning year 2, daily oral ursodiol is administered to all patients. Patients with disease progression are crossed to the alternate group or undergo liver transplantation if clinically indicated.


Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


--Disease Characteristics-- Biopsy proven primary biliary cirrhosis (PBC); Disproportionate increase in alkaline phosphatase; Positive antimitochondrial antibody test OR Symptoms consistent with PBC, e.g.: pruritus, fatigue, malaise, jaundice, elevated bilirubin

No clinically advanced PBC, i.e.: bilirubin greater than 10 mg/dL or albumin less than 2.5 g/dL, determined by 2 analyses 10 weeks apart; bleeding esophageal varices or congestive gastropathy; chronic hepatic encephalopathy; chronic ascites

--Prior/Concurrent Therapy-- No concurrent drugs associated with chronic liver disease

--Patient Characteristics--

Hematopoietic: WBC at least 2500 Platelets at least 100,000 (unless due to hypersplenism); Hematocrit at least 30%

Renal: No renal disease that could cause liver dysfunction

Other: No history of alcohol abuse; No other medical illness that might cause liver dysfunction, e.g., severe cardiac failure; No pregnant women

  Contacts and Locations
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Please refer to this study by its identifier: NCT00004748

Sponsors and Collaborators
National Center for Research Resources (NCRR)
Tufts Medical Center
Study Chair: Marshall M. Kaplan Tufts Medical Center
  More Information Identifier: NCT00004748     History of Changes
Other Study ID Numbers: 199/11664  NEMCH-454 
Study First Received: February 24, 2000
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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

Additional relevant MeSH terms:
Liver Cirrhosis
Liver Cirrhosis, Biliary
Pathologic Processes
Liver Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Ursodeoxycholic Acid
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Physiological Effects of Drugs
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Dermatologic Agents
Enzyme Inhibitors
Folic Acid Antagonists
Immunosuppressive Agents
Immunologic Factors
Antirheumatic Agents
Nucleic Acid Synthesis Inhibitors
Gout Suppressants
Tubulin Modulators processed this record on October 25, 2016