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.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Research Project--Cooperative Agreements (U01)
Project #
Application #
Study Section
National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
Program Officer
Odenkirchen, Joanne
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
University of Alabama Birmingham
Biostatistics & Other Math Sci
Schools of Public Health
United States
Zip Code
Sengupta, Manjistha; Wang, Bi-Dar; Lee, Norman H et al. (2018) MicroRNA and mRNA expression associated with ectopic germinal centers in thymus of myasthenia gravis. PLoS One 13:e0205464
Weis, Cleo-Aron; Aban, Inmaculada B; Cutter, Garry et al. (2018) Histopathology of thymectomy specimens from the MGTX-trial: Entropy analysis as strategy to quantify spatial heterogeneity of lymphoid follicle and fat distribution. PLoS One 13:e0197435
Wolfe, Gil I; Kaminski, Henry J; Aban, Inmaculada B et al. (2016) Randomized Trial of Thymectomy in Myasthenia Gravis. N Engl J Med 375:511-22
Aban, Inmaculada B; George, Brandon (2015) Statistical considerations for preclinical studies. Exp Neurol 270:82-7
Kaminski, Henry J; Kusner, Linda L; Wolfe, Gil I et al. (2012) Biomarker development for myasthenia gravis. Ann N Y Acad Sci 1275:101-6
Minisman, Greg; Bhanushali, Minal; Conwit, Robin et al. (2012) Implementing clinical trials on an international platform: challenges and perspectives. J Neurol Sci 313:1-6
Kister, Ilya; Chamot, Eric; Bacon, Joshua H et al. (2011) Trend for decreasing Multiple Sclerosis Severity Scores (MSSS) with increasing calendar year of enrollment into the New York State Multiple Sclerosis Consortium. Mult Scler 17:725-33
Marrie, Ruth Ann; Cutter, Gary; Tyry, Tuula (2011) Substantial adverse association of visual and vascular comorbidities on visual disability in multiple sclerosis. Mult Scler 17:1464-71
Mandawat, Aditya; Kaminski, Henry J; Cutter, Gary et al. (2010) Comparative analysis of therapeutic options used for myasthenia gravis. Ann Neurol 68:797-805
Marrie, R A; Rudick, R; Horwitz, R et al. (2010) Vascular comorbidity is associated with more rapid disability progression in multiple sclerosis. Neurology 74:1041-7

Showing the most recent 10 out of 15 publications