Steroids and Methotrexate to Treat Systemic Vasculitis
Status:
Completed
Trial end date:
2004-02-01
Target enrollment:
Participant gender:
Summary
This study will evaluate the safety and effectiveness of prednisone and methotrexate in
treating severe Wegener's granulomatosis and other systemic vasculitides. These diseases
involve inflammation of blood vessels (vasculitis) that may affect the brain, nerves, eyes,
sinuses, lungs, kidneys, intestinal tract, skin, joints, heart and other sites. Current
treatment with prednisone and the anti-cancer drug cyclophosphamide is effective, but has
significant side effects and a high rate of disease recurrence. In a small number of patients
with vasculitis, prednisone and methotrexate, another anti-cancer drug, have led to marked
improvement, with fewer side effects than are seen with cyclophosphamide. This study will
evaluate this drug combination in a larger patient population.
Patients 10 to 80 years of age with active Wegener's granulomatosis, polyarteritis nodosa,
Churg-Strauss vasculitis, or microscopic polyangiitis overlap may be eligible for this 2 1/2
to 3-year study. In addition, patients with glomerulonephritis (a type of kidney disease) and
a positive blood test for C-ANCA (antibodies found in certain vasculitic kidney diseases) or
inflammatory sinusitis or lung nodule or infiltrates in the absence of infection may also be
enrolled.
Participants will take prednisone daily, by mouth, and low-dose methotrexate weekly, by mouth
or by injection either under the skin, into a muscle or into a vein. Patients who
significantly improve with treatment will gradually reduce, and eventually stop, the
prednisone. If the remission lasts, methotrexate will also be reduced and stopped after 2 1/2
years. If active disease recurs, the original treatment program may be started again.
Patients who never achieve complete remission with treatment but whose symptoms are well
controlled and experience no serious side effects may choose to either continue low-dose
methotrexate or stop therapy.
Patients will be hospitalized 4 to 6 times a year, about 2 to 8 days each time, depending on
their disease severity and response to illness. In addition, they will have the following
tests and procedures:
- Medical history and physical examination (upon admission to the study and then every 1
to 3 months).
- Blood tests for blood cell counts and for levels of enzymes that indicate liver damage
(upon admission, then weekly, and finally, no less than monthly).
- Additional blood tests to measure blood chemistries and evaluate kidney function (upon
admission and again when clinically indicated).
- Chest X-rays (upon admission and when clinically indicated).
- Computerized tomography (CT) and magnetic resonance imaging (as needed).
- Electrocardiogram (upon admission and then as clinically indicated).
- Lung function studies (upon admission and at least every 6 months or as clinically
indicated).
- Ear, nose and throat evaluations (as clinically indicated).
- Liver biopsy, if blood tests to monitor liver function are persistently abnormal. This
procedure is done in the hospital under sedation to induce relaxation and drowsiness.
The skin over the liver (upper right abdomen) is numbed with a local anesthetic and a
needle is passed rapidly in and out of the liver to collect a small tissue sample for
microscopic examination.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)