Thymectomy in Non-Thymomatous MG Patients on Prednisone

描述（由申请方提供）：MGTX是一项多中心、单盲、随机研究，在接受泼尼松治疗的非胸腺瘤型重症肌无力（MG）患者中比较胸腺切除术与非胸腺切除术。这个由NINDS赞助的项目于2005年9月启动。招募目标为200例受试者，其中100例接受胸腺切除术加泼尼松治疗，100例接受泼尼松单药治疗。本申请旨在更新该补助金，以将招募期再延长两年，并在2012年11月前完成招募，并对患者进行三年随访。主要结局和其他结局指标与原始方案相同。计划继续招募和随访所有随机化受试者，保持盲态，直至最后一名入组受试者完成所有3年研究。该计划与研究DSMB以及研究者批准的原始资助一致，但现在对一些患者进行了更长时间的随访（长达6年）。对于此次更新，我们在下文提供了最初的具体目标、研究设计的详细信息、研究进展报告（包括招募结果和迄今为止加强招募的策略）、研究核心组成部分的详细信息（临床协调中心、数据协调中心和MG研究中的相关生物标志物）、所有管理活动的详细信息、研究人群的基线人口统计学资料，数据的数量和质量、完成研究的时间轴、当前MG治疗竞技场的概述及其对本研究的影响。我们建议对原始研究进行的唯一更改是将样本量从200减少到150。最初的样本量计算被夸大，以确保足够的样本量用于预期的脱落和审查员提出的亚组假设。此次对150名受试者的修订是基于对我们招募能力的现实估计，该能力来自三年的招募经验，并且总共150名患者（本请求中增加了70名）仍然足以满足我们的主要具体目标，并且可以在本预算请求内完成。 公共卫生相关性：这项研究所解决的问题已经挑战了MG社区超过50年，这项研究很可能是最后一次组织和/或完成这样的试验。

DESCRIPTION (provided by applicant): The MGTX is a multi-center, single-blind, randomized study comparing thymectomy to no thymectomy in non-thymomatous myasthenia gravis (MG) patients receiving prednisone. This NINDS-sponsored project was initiated in September, 2005. The recruitment goal has been 200 subjects -100 to thymectomy plus prednisone and 100 to prednisone alone. This application is for renewal of this grant to extend the recruitment period for an additional two years and complete recruitment by November 2012 and follow patients for three years. The primary outcome and other outcome measures are unchanged from the original protocol. The plan is to continue recruitment and follow-up of all subjects, as randomized, with blinding being maintained until the last enrolled subject has completed all three years of the study. This plan is consistent from the original grant as approved by the study DSMB as well as the investigators, but will now have a longer follow-up for some patients (out to 6 years). For this renewal, we provide below the original specific aims, details of the study design, a study progress report including recruitment results and strategies to enhance recruitment to date, details of the study core components (Clinical Coordinating Center, Data Coordinating Center, and the associated Biomarker in MG study), details of all administrative activities, the baseline demographics of the study population, volume and quality of the data, a timeline for the completion of the study, and an overview of the current MG therapeutic arena and its impact on this study. The only changes we propose from the original study are to reduce the sample size from 200 to 150. The original sample size calculations were inflated to insure adequate sample sizes for anticipated drop-outs and subgroup hypotheses raised by the reviewers. This revision to 150 subjects is based on realistic estimates of our ability to recruit derived from a three-year experience of enrollment and the fact that the 150 total patients (70 additional in this request) remains adequate for our primary specific aim and can be completed within this budget request. PUBLIC HEALTH RELEVANCE: The question being addressed by this study has challenged the MG community for over 50 years and this study is most likely the last time such a trial will be organized and/or completed.