This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. OBJECTIVE: The goal of this study is to test the hypothesis that MM (2.5 Grams /day) in combination with prednisone (20 mg/d) provides better control of myasthenic signs and symptoms than prednisone alone in the initial management of MG. An open label extension will supply further information about the safety and tolerability of MM at does ranging from 2.5 to 3 grams/day and will give preliminary information about tapering and discontinuing prednisone in patients maintained on MM. RESEARCH PLAN: This is a randomized, double-blind, placebo-controlled, multicenter, trial of MM with moderate-dose prednisone in patients whose MG is not severe. Double blind treatment is three (3) months followed by a 6 month open late phase. Expected multicenter enrollment is 80 patients. METHODS: Subjects will be randomized to receive prednisone 20 mg /day with placebo or with MM 2.5 g/d. Blood work and ADL questionnaire will be administered to assure safety and detect initial improvement. Safety evaluation and assessment of disease severity will occur after 4, 8, and 12 weeks on study medication. Open Label will continue for with safety evaluation and assessments at 13, 14, 15, 16, 20, 28 and 36 weeks. CLINICAL
MG is an uncommon autoimmune disease, affecting about 50,000 patients in the US, in which antibodies to the acetylcholine receptor cause failure of neuromuscular transmission, pathologic fatigue, and weakness. Immunosuppression is used in most MG patients but current treatments are not uniformly effective and carry risk of serious side effects. Preliminary reports have suggested that mycophenolate mofetil (MM), a selective inhibitor of T amp; B-cells used to prevent allograft rejection, is effective and well tolerated in MG. A major advantage of MM, compared to the steroid-sparing agents currently in use, is the rapid onset (within 6 weeks) of its anti-myasthenic action, raising the possibility that initial treatment with MM will lessen the duration/dose of daily corticosteroids and reduce the burden of corticosteroid-related toxicity. This study will furnish key data about the safety and efficacy of MM in MG and represents an important step toward the goal of organ-specific, corticosteroid-free immunosuppressive treatment for this diseas
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