Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004748
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : June 24, 2005
Tufts Medical Center
Information provided by:
Office of Rare Diseases (ORD)

February 24, 2000
February 25, 2000
June 24, 2005
November 1989
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Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis


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.


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.

Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Liver Cirrhosis, Biliary
  • Drug: colchicine
  • Drug: methotrexate
  • Drug: ursodiol
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
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--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

Sexes Eligible for Study: All
Child, Adult, Older Adult
Contact information is only displayed when the study is recruiting subjects
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National Center for Research Resources (NCRR)
Tufts Medical Center
Study Chair: Marshall M. Kaplan Tufts Medical Center
Office of Rare Diseases (ORD)
December 2001

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP