Thymectomy in Non-Thymomatous MG Patients on Prednisone

接受泼尼松治疗的非胸腺瘤型重症肌无力患者的胸腺切除术

• 批准号: 8526574
• 负责人: GARY R CUTTER
• 金额: $ 97.65万
• 依托单位: UNIVERSITY OF ALABAMA AT BIRMINGHAM
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-09-23 至 2015-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8526574
• 关键词: Address; Biological Markers; Budgets; Clinical; Communities; Data Coordinating Center; Data Quality; Drops; Enrollment; Goals; Grant; Myasthenia Gravis; National Institute of Neurological Disorders and Stroke; Outcome Measure; Outpatients; Patients; Population Study; Prednisone; Progress Reports; Protocols documentation; Randomized; Recruitment Activity; Research Design; Research Personnel; Sample Size; Subgroup; Therapeutic; Thymectomy; Time; TimeLine; base; blind; demographics; experience; follow-up; primary outcome; public health relevance

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.

描述（由申请人提供）：MGTX 是一项多中心、单盲、随机研究，比较接受泼尼松治疗的非胸腺瘤性重症肌无力 (MG) 患者的胸腺切除术和不切除胸腺术。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 名接受胸腺切除术加泼尼松，100 名接受单独泼尼松。本申请旨在续签该补助金，将招募期再延长两年，并于 2012 年 11 月之前完成招募并跟踪患者三年。主要结局和其他结局指标与原始方案相比没有变化。该计划是继续随机招募和随访所有受试者，并维持盲法，直到最后一名受试者完成全部三年的研究。该计划与 DSMB 和研究人员批准的最初拨款一致，但现在将对某些患者进行更长的随访（长达 6 年）。对于本次更新，我们在下面提供了最初的具体目标、研究设计的详细信息、研究进度报告，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 研究中相关的生物标志物）、所有管理活动的详细信息、研究人群的基线人口统计、数据的数量和质量、完成研究的时间表以及当前 MG 治疗领域及其影响的概述这项研究。我们建议对原始研究的唯一改变是将样本量从 200 个减少到 150 个。原始样本量计算被夸大，以确保有足够的样本量来满足预期的退出和评审者提出的亚组假设。此次对 150 名受试者的修订是基于我们根据三年的入组经验对招募能力的实际估计，以及 150 名患者总数（本请求中另外 70 名）仍然足以满足我们的主要具体目标并且可以完成的事实在此预算要求内。