Mycophenolate Mofetil to Treat Wegener's Granulomatosis and Related Vascular Inflammatory Conditions
Status:
Completed
Trial end date:
2004-06-01
Target enrollment:
Participant gender:
Summary
This study will examine the safety and effectiveness of the drug mycophenolate mofetil (MPM)
in treating Wegener's granulomatosis and related inflammatory vessel diseases. Blood vessel
inflammation in these patients may involve different parts of the body, including the brain,
nerves, eyes, sinuses, lungs, kidneys, intestinal tract, skin, joints, heart, and other
sites. The more severe the involvement, the more likely the disease will be life-threatening.
Standard treatment consists of combination drug therapy with prednisone and a cytotoxic
agent-usually cyclophosphamide or methotrexate. However, some patients in whom this treatment
is initially successful have a disease relapse; other patients cannot take the medications
because of other health problems or because of severe side effects of the drugs.
MPM is approved by the Food and Drug Administration to prevent kidney transplant rejection.
It is chemically similar to another cytotoxic drug called azathioprine, which has been
beneficial in maintaining remission in patients with Wegener's granulomatosis who have been
treated successfully with cyclophosphamide. Because MPM is more effective than azathioprine
in preventing organ rejection, it may also prove beneficial as a second-line treatment for
Wegener's granulomatosis.
Patients with Wegener's granulomatosis or related inflammatory vessel diseases who have had a
relapse following treatment with cyclophosphamide and methotrexate or who cannot take one or
both of these drugs may be eligible for this study. Only patients who have been treated at
NIH in the methotrexate protocol or the cyclophosphamide switching to methotrexate protocol,
or who have received the exact same treatment from their own physician may participate.
Participants will have a complete medical evaluation including laboratory studies.
Consultations, X-rays and biopsies of affected organs may also be done if indicated for
diagnosis or treatment. Patients with active disease will be given MPM and prednisone, both
in tablet form. Patients with inactive disease will receive only prednisone if they are
already taking it. In both cases, the prednisone will be reduced gradually and discontinued
if the disease improves significantly. MPM therapy will continue for at least 2 years. If
after 2 years the disease remains in remission, the MPM dose will be gradually reduced and
then stopped. If active disease recurs while on MPM therapy, the treatment plan will likely
be changed. The new regimen will be determined by the severity of disease, other medical
conditions, and history of side effects to previous medications.
Patients will be followed at the NIH clinic every month for the first 3 months on MPM and
then every 3 months for another 18 months. Those whose disease has remained in remission and
have stopped all medications will then be followed every 6 months for 4 visits. The follow-up
visits will include a physical examination, blood draws, and, if needed, X-rays. Visits may
be scheduled more frequently if medically indicated.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)