Prednisone Ocular Myasthenia Gravis Trial Planning Grant

描述（由申请人提供）：重症肌无力是一种慢性自身免疫性神经肌肉疾病，其特征是肌肉无力，在活动期间加重，休息后改善，可导致危及生命的并发症，如呼吸衰竭。眼重症肌无力通常表现为眼部肌肉无力，但在1至2年内，约三分之二的患者发展为全身性疾病。本申请旨在为眼部重症肌无力患者临床试验的最终计划提供资金支持。一项包含240名患者的双盲、安慰剂对照、随机临床试验将被设计来检验这样一种假设：在不引起重大不良事件或副作用的剂量下，皮质类固醇治疗强的松将减少全身肌肉受累的发生率，并在一年内改善疾病相关的视力障碍。降低全身性重症肌无力的风险将降低整体残疾、重症肌无力危象、住院和昂贵的侵入性治疗（如胸腺切除术和血浆导入）的风险。此外，如果建议的治疗减少复视和恢复双眼功能，患者可能能够更好地进行日常生活活动，如阅读、驾驶汽车、操作机械和工作。如果慢性低剂量皮质类固醇有利于降低全面性疾病的发生率和减轻视力障碍，它将取代许多神经科医生使用的中至高剂量治疗；避免了高血压、感染、高血糖和骨质疏松等并发症的发生。在为期一年的奖励期间，我们的活动将通过执行委员会会议和电话会议，指导制定程序手册，最终确定研究方案，招募诊所地点，概述数据管理系统，详细说明研究药物分配和跟踪，创建诊所和患者手册，准备和提交研究试验的资助申请。

DESCRIPTION (provided by applicant): Myasthenia gravis, a chronic autoimmune neuromuscular disease, is characterized by muscle weakness that increases during periods of activity and improves after periods of rest and can result in life-threatening complications such as respiratory failure. Often patients present with weakness confined to the ocular muscles, Ocular Myasthenia Gravis but within 1 to 2 years, approximately two-thirds of patients develop generalized disease. This application is for funding to support the final planning of a clinical trial for patients with Ocular Myasthenia Gravis. A double masked, placebo-controlled, randomized clinical trial of 240 patients will be designed to test the hypothesis that corticosteroid therapy with prednisone, in doses that do not cause major adverse events or side effects, will reduce the incidence of systemic muscle involvement and improve disease-related visual disability at one year. Reducing the risk of Generalized Myasthenia Gravis will decrease the risk of overall disability, Myasthenic crises, hospitalizations, and costly invasive therapies such as thymectomy and plasmaphoresis. In addition, if the proposed therapy reduces double vision and restores binocular function, patients may be better able to perform activities of daily living such as reading, driving an automobile, handling machinery, and work. If chronic low dose corticosteroids are beneficial in decreasing the incidence of generalized disease as well as reducing visual disability, it will replace the moderate to high dose therapy used by many neurologists; there by avoiding the significant complications of hypertension, infections, hyperglycemia, and osteoporosis. Our activities during the one year award period will be directed towards developing a Manual of Procedures, finalizing Study protocol, recruiting clinic sites, outlining data management systems, detailing Study drug distribution and tracking, creating clinic and patient brochures, and preparing and submitting the grant applications for the Study trial, via executive committee meetings and teleconferences.