Ignite Creation Date:
2024-05-05 @ 11:07 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001764
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
Mycophenolate Mofetil to Treat Wegeners Granulomatosis and Related Vascular Inflammatory Conditions
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Mycophenolate Mofetil in the Treatment of Wegeners Granulomatosis and Related Vasculitides
Status:
COMPLETED
Status Verified Date:
2004-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will examine the safety and effectiveness of the drug mycophenolate mofetil MPM in treating Wegeners 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 Wegeners 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 Wegeners granulomatosis
Patients with Wegeners 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
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 Wegeners 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 Wegeners granulomatosis
Patients with Wegeners 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
Detailed Description:
The purpose of this study is to assess the efficacy and safety of mycophenolate mofetil in the treatment of Wegeners granulomatosis and related vasculitides in patients who have contraindications to methotrexate or cyclophosphamide or have experienced disease relapse while on these agents Mycophenolate mofetil is a novel immunosuppressive agent which has been approved by the FDA for renal transplantation It is chemically similar to and has a potentially greater efficacy in preventing acute transplant rejection than azathioprine a drug which has been previously used as an alternative treatment for vasculitis In this study patients who have had disease relapse or contraindications to methotrexate will initially receive cyclophosphamide and glucocorticoids and then switch from cyclophosphamide to mycophenolate mofetil upon disease remission If at the end of two years of mycophenolate mofetil therapy there is continued evidence of disease remission the drug will be tapered and discontinued Patients with contraindications to both cyclophosphamide and methotrexate will be treated initially with prednisone and mycophenolate mofetil with mycophenolate mofetil being continued for two full years after disease remission and then tapered and discontinued For patients who develop intolerance to methotrexate or cyclophosphamide while their disease is in remission mycophenolate mofetil will be started and continued for two years after which time it will be tapered and discontinued Patients will be prospectively monitored for evidence of disease activity 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 effects
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 98-I-0092 | None | None | None |