Phase 1-2 Pilot Study of Rituximab (Rituxan) in Refractory Myasthenia Gravis.
Myasthenia gravis (MG) is an immune-mediated disorder of the neuromuscular junction
diagnosed on the basis of clinical, electrophysiological and serological features.
Cyclosporine as a disease-modifying therapy has been effective in a controlled study;
corticosteroids, immunosuppressive agents such as azathioprine and cyclophosphamide,
plasmapheresis and intravenous human immune globulin have shown benefit in uncontrolled
trials. There are several drawbacks to currently used medical treatments, including serious
and debilitating side-effects, prohibitive costs, and the need for continuous or periodical
treatment. Almost 20-25% of patients with MG are unresponsive to commonly used therapies,
resulting in significant burden and economic loss. Rituximab is a chimeric anti-CD20
monoclonal antibody which produces a substantial reduction in circulating plasma cells
(CD19+) and B cells (CD20+) and provides targeted therapy for B-cell lymphomas. Recently,
rituximab has been found to be effective in several antibody-mediated autoimmune processes,
including immune thrombocytopenia, autoimmune hemolytic anemia, and IgM-related
polyneuropathies. There is preliminary evidence in the literature that treatment of MG
patients with rituximab is likely to be of benefit. These observations would strongly
suggest that rituximab might benefit refractory MG and needs further study.
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To examine the effects of rituximab on disease activity in MG patients with refractory disease.
Patients will be followed for one year
Rup Tandan, MD, FRCP
University of Vermont Department of Neurology
United States: Food and Drug Administration
|State University of New York||Buffalo, New York 14203-1154|
|University of Vermont Department of Neurology||Burlington, Vermont 05405|