Thymectomy in Non-Thymomatous MG Patients on Prednisone

描述(申请人提供)：MGTX是一项多中心、单盲、随机研究，比较接受强的松治疗的非胸腺瘤性重症肌无力(MG)患者胸腺切除和不胸腺切除的疗效。这个由NINDS赞助的项目于2005年9月启动。招募目标是200名受试者-100名胸腺切除加强的松治疗，100名单用强的松治疗。这项申请是为了续期，将招募期限再延长两年，并在2012年11月之前完成招募，并对患者进行三年的跟踪调查。主要成果和其他成果衡量标准与原始议定书保持不变。该计划将继续以随机方式招募和跟踪所有受试者，并保持失明，直到最后一名受试者完成所有三年的研究。这项计划与最初的拨款是一致的，由研究dsmb和研究人员批准，但现在对一些患者将有更长的随访时间(长达6年)。对于这次更新，我们提供了以下最初的具体目标、研究设计的细节、包括招募结果和迄今加强招募的策略的研究进展报告、研究核心组成部分的细节(临床协调中心、数据协调中心和MG研究中的相关生物标记物)、所有管理活动的细节、研究人群的基线人口统计学、数据的数量和质量、研究完成的时间表、以及当前MG治疗领域及其对本研究的影响的概述。我们从最初的研究中提出的唯一变化是将样本量从200减少到150。最初的样本量计算被夸大，以确保评价者提出的预期辍学和分组假设有足够的样本量。这次对150名受试者的修订是基于对我们招募能力的现实估计，这源于三年的登记经验，以及这样一个事实，即总共150名患者(在本申请中增加了70名)仍然足以满足我们的主要具体目标，并可以在这一预算要求内完成。

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.