Comparison of Treatments to Maintain Disease Remission in Patients With Wegener’s Granulomatosis and Related Vasculitis Syndromes

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Brief Title

Comparison of Treatments to Maintain Disease Remission in Patients With Wegener's Granulomatosis and Related Vasculitis Syndromes

Official Title

A Randomized Trial Comparing Methotrexate Versus Mycophenolate Mofetil for Remission Maintenance in Wegener's Granulomatosis and Related Vasculitides

Brief Summary

      This study will compare the safety and effectiveness of two drugs-methotrexate and
      mycophenolate mofetil (MPM)-in preventing disease recurrence in patients with Wegener's
      granulomatosis and related inflammatory blood vessel disorders. The standard treatment for
      these conditions is combination drug therapy with prednisone plus cyclophosphamide. However,
      although most patients improve on this therapy and achieve disease remission, many experience
      a relapse (return of the disease) some time after therapy is stopped. Also, these drugs can
      produce serious side effects during treatment. This study will test a new treatment regimen
      to try to maintain disease remission in these patients with minimal side effects.

      Patients with Wegener's granulomatosis or other related blood vessel disorders between 10 and
      80 years old will be considered for this study. All participants will start therapy with
      daily doses of prednisone and cyclophosphamide. Prednisone will be reduced gradually and then
      stopped after symptoms improve significantly. Cyclophosphamide will continue until the
      disease is in remission. Patients in remission will then be randomly assigned to continue
      treatment with either MPM or methotrexate. MPM is taken twice a day by mouth. Methotrexate is
      taken once a week, usually by mouth, but in some cases, by injection into a muscle or under
      the skin. Patients who do well and have no side effects will continue treatment for 2 years.
      Then, the drug will gradually be reduced (usually at monthly intervals) and finally stopped.
      No further treatment will be given unless a relapse occurs. At that time, the type of
      treatment will depend on various medical factors, including the severity of the recurrence
      and the patient's history of drug side effects.

      Physical examinations and various tests, including blood and urine analyses, and X-rays, will
      be done periodically to evaluate the response to treatment and monitor drug side effects. The
      total duration of the study-from the screening evaluation through a 2-year follow up after
      all medications have been stopped-is about 5 to 6 years.

Detailed Description

      The purpose of this study is to assess the comparative efficacy of using methotrexate versus
      mycophenolate mofetil for maintaining remission that has been induced by cyclophosphamide and
      glucocorticoids in patients with Wegener's granulomatosis and related vasculitides. In this
      study, all patients will initially receive daily cyclophosphamide and glucocorticoids and
      then at disease remission, cyclophosphamide will be discontinued and patients will be
      randomized to receive either methotrexate or mycophenolate mofetil for remission maintenance.
      They will continue to receive the agent to which they are randomized for 2 years, after which
      time it will be tapered and discontinued. Patients will be prospectively monitored for
      evidence of disease relapse and drug toxicity. Specific parameters that will be obtained
      include the time to disease remission, the rate and time of disease relapse, and the
      incidence of drug-related adverse events.

Study Phase

Phase 2

Study Type





Mycophenolate Mofetil


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

February 2000

Completion Date

June 2004

Eligibility Criteria


          1. Documentation of Wegener's granulomatosis (WG) or a related systemic vasculitis based
             on clinical characteristics and histopathologic and/or angiographic evidence of
             vasculitis. In the absence of histopathologic and/or angiographic evidence of
             vasculitis, patients who meet one of the following criteria and in whom infectious and
             autoimmune diseases that may mimic WG or a related systemic vasculitides have been
             excluded will also be eligible:

             A. A positive assay for anti-neutrophil cytoplasmic autoantibodies (C- or P-ANCA) and
             the presence of glomerulonephritis defined by red blood cell casts and proteinuria or
             renal biopsy showing necrotizing glomerulonephritis in the absence of immune deposits.

             B. A positive assay for anti-neutrophil cytoplasmic autoantibodies (C- or P-ANCA) and
             the presence of granulomatous inflammation on biopsy plus abnormal chest radiograph
             (defined as the presence of nodules, fixed infiltrates, or cavities) plus nasal/oral
             inflammation on clinical examination.

          2. Age 10-80 years.

          3. Evidence of active disease as defined by a Vasculitis Disease Activity Index of
             greater than or equal to 3 or if begun on CYC and glucocorticoid at an outside
             institution, a history of a Vasculitis Disease Activity Index greater than or equal to
             3 at the time of therapy initiation.


          1. Evidence of active infection which, in the judgment of the investigator, is of greater
             danger to the patient than the underlying vasculitis. In those instances in which
             infection cannot be ruled out by gram stain and culture of secretions or collections
             of fluid in involved organs, it may be necessary to obtain a biopsy of the affected
             tissue for microbiological and histopathological studies.

          2. Patients who are pregnant or who are nursing infants will not be eligible. Fertile
             women must have a negative pregnancy test within one week prior to study entry and
             must be using an effective means of birth control.

          3. Serological evidence of infection with human immunodeficiency virus, hepatitis C, or a
             positive hepatitis B surface antigen. A serological determination will be performed
             within two weeks of beginning study participation.

          4. Acute or chronic liver disease, past history of alcohol abuse (greater than 14 oz of
             100 proof liquor or equivalent per week), ongoing alcohol use of any volume that
             cannot be discontinued upon entry into the study.

          5. History of CYC- or methotrexate- induced pneumonitis with past treatment.

          6. Hypersensitivity to CYC, MPM, or methotrexate.

          7. Transitional cell carcinoma of the bladder.

          8. Inability to comply with study guidelines.

          9. Hemocytopenia: platelet count less than 80,000/mm(3), leukocyte count less than
             3,000/mm(3), hematocrit less than 20% (in the absence of gastrointestinal bleeding or
             hemolytic anemia).




N/A - N/A

Accepts Healthy Volunteers



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Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Study Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Study Sponsor

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Verification Date

June 2004