Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy

A Multi-Center, Single-Blind, Randomized Study Comparing Thymectomy to No Thymectomy in Non-Thymomatous Myasthenia Gravis (MG) Patients Receiving Prednisone

The purpose of this trial is to determine if thymectomy combined with prednisone therapy is more beneficial in treating non-thymomatous myasthenia gravis than prednisone therapy alone.

Study Overview

• Status: Completed
• Conditions: Myasthenia Gravis
• Intervention / Treatment: Procedure: thymectomy plus prednisone; Drug: prednisone alone

Detailed Description

Myasthenia gravis (MG) is an autoimmune disease involving the thymus in which 85 percent of patients have antibodies to muscle acetylcholine receptors (AchR-Ab) that interfere with neuromuscular transmission. MG frequently causes severe disability that can be life-threatening. Thymectomy-a surgical procedure that removes thymus gland tissue from the chest cavity-has been an established therapy for non-thymomatous MG, or MG without thymoma, for more than 60 years (based on retrospective, non-randomized studies). Corticosteroids are now being used increasingly either as the sole treatment or in combination with thymectomy. Both therapies have associated adverse effects and indications for their use based on randomized trial data are lacking.

The purpose of this 5-year trial is to determine if the surgical procedure, extended transsternal thymectomy (ETTX), combined with prednisone therapy is more beneficial in treating individuals with non-thymomatous MG than prednisone therapy alone. More specifically, this study will determine 1) if ETTX combined with prednisone results in a greater improvement in myasthenic weakness, compared to prednisone alone; 2) if ETTX combined with prednisone results in a lower total dose of prednisone, thus decreasing the likelihood of concurrent and long-term toxic effects, compared to prednisone alone; and 3) if ETTX combined with prednisone enhances quality of life by reducing adverse events and symptoms associated with the therapies, compared to prednisone alone.

Learning that thymectomy results in a meaningful reduction of prednisone dosage or even full withdrawal or reduces side effects related to prednisone would support using the two treatments-thymectomy and prednisone-together. However, if no meaningful reduction of prednisone dosage or side effects is shown, the results would mean that using the two treatments together offers no advantages over prednisone treatment alone.

After an initial screening, study participants will be randomized either to undergo the surgical procedure ETTX and receive prednisone treatment, or to receive prednisone treatment alone without surgery. Participants will be followed for at least 3 years.

• Study Type: Interventional
• Enrollment (Actual): 126
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina
    • University of Buenis, Centro de Asistencia Docencia e Investigacion en Miastenia (CADIMI) Av. Forest 1146 - Ciudad Autonoma de Buenos Aires
• Australia
  • Sydney, Australia
    • University of Sydney, Royal Prince Alfred Hospital and The University of Sydney
  • Victoria, Australia, 3050
    • University of Melbourne, Melbourne, The Royal Melbourne Hospital, Dept of Neurology, Royal Melbourne Hospital
• Brazil
  • Brasilia, Brazil, CEP 71640 255
    • Hospital de Base do Distrito Federal
  • Curitiba, Brazil, 80060-900
    • Universidade Federal do Parana
  • Rio De Janeiro, Brazil, CEP 20520-053
    • Federal University of Rio de Janeiro
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 2T9
      • University of Calgary, Heritage Medical Research Clinic Room 1132 3330 Hospital Dr NW
  • British Columbia
    • Vancouver, British Columbia, Canada, V6T 2B5
      • University of British Columbia
  • Ontario
    • Ottawa, Ontario, Canada, K1H 8L6
      • University of Ottawa, The Ottawa Hospital General Campus, Division of Neurology, 501 Smyth Rd. Box 601
  • Quebec
    • Montreal, Quebec, Canada, H3G 1A4
      • McGill University Health Center
• Chile
  • Santiago, Chile
    • Hospital Del Salvador, Departamento de Ciencias Neurológicas, Universidad de Chile, Salvador 95 Of 416, Providencia
• Germany
  • Münster, Germany, 48149
    • University of Münster, Schlossplatz 2
  • Tübingen, Germany, 72076
    • University of Tubingen
  • Baden-Württemberg
    • Mannheim, Baden-Württemberg, Germany, 69117
      • University of Heidelberg, Seminarstraße 2
  • Bavaria
    • Regensburg, Bavaria, Germany, 93043
      • University of Regensburg, Dept. of Neurology, Universitätsstr. 84, D
  • North Rhine-Westphalia
    • Düsseldorf, North Rhine-Westphalia, Germany, D-40225
      • University of Düsseldorf
  • Rhineland-Palatinate
    • Mainz, Rhineland-Palatinate, Germany, 55101
      • Johannes-Gutenberg University, Klinikum der Johannes Gutenberg-Universität, Klinik und Poliklinik für Neurologie, Langenbeckstr
• Italy
  • Milan, Italy, 20133
    • National Neurological Institute "Carlo Besta", Myopathology and Immunology Unit, Dept of Neurology IV, Natl. Neurolog Inst. "C. Besta", Via Celoria, 11,
  • Rome, Italy, 00189
    • University of Rome "Sapienza"
  • Rome, Italy
    • Catholic University, Universita Cattolica del Sacro Cuore, Largo Agostino Gemelli 8, e
  • Torino, Italy
    • University of Torino
• Japan
  • Ishikawa
    • Kanazawa, Ishikawa, Japan, 920-8641
      • Kanazawa University, Department of Neurology, Kanazawa University Hospital, 13-1 Takaramachi
  • Kyushu
    • Nagasaki, Kyushu, Japan, 852-8501
      • Nagasaki University, First Department of Internal Medicine,Graduate School of Biomedical Sciences,1-7-1,Sakamoto
• Mexico
  • Mexico, Mexico, 14000
    • Instituto Nacional de la Nutricion
• Netherlands
  • Leiden, Netherlands
    • Leiden University
• Poland
  • Województwo
    • Warsaw, Województwo, Poland, 02 097
      • Medical University of Warsaw
    • Warsaw, Województwo, Poland
      • Institute of Tuberculosis and Lung Disease
• Portugal
  • Porto, Portugal, 4099-001
    • Porto University, Serviço de Neurologia,Hospital Geral de Santo António, Largo Prof Abel Salazar
• South Africa
  • Cape Town, South Africa
    • University of Cape Town, Division of Neurology E8-74, Groote Schuur Hospital,Observatory
• Spain
  • Barcelona, Spain, 08025
    • H. Sant Pau, Universitat Autònoma de Barcelona, Neurology Department, Hospital Sta Creu i Sant Pau, C/Mas Casanovas no 90 4o pis 4o modul.
• Taiwan
  • New Taipei, Taiwan, 24205
    • Fu-Jen Catholic University, No. 510, Zhongzheng Rd., Xinzhuang Dist
• Thailand
  • Bangkok, Thailand, 10400
    • Ramathibodi Hospital, Mahidol University
• United Kingdom
  • Glasgow, United Kingdom, G51 4TF
    • Queen Elizabeth University Hospital, Glasgow
  • Liverpool, United Kingdom, L9 7LJ
    • Walton Centre for Neurology and Neurosurgery, Liverpool Heart and Chest Hospital, Liverpool. The Walton Centre for Neurology and Neurosurgery, Lower Lane, Fazakerley
  • Manchester, United Kingdom, M13 9PL
    • University of Manchester, Oxford Road
  • Oxford, United Kingdom, OX2 6HE
    • University of Oxford, Dept of Clinical Neurology, University of Oxford, Radcliffe Infirmary
  • Sheffield, United Kingdom, S10 2TN
    • University of Sheffield, Western Bank
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham, Department of Neurology, Sparks Center, Suite 350, 1720 7th Avenue South
    • Birmingham, Alabama, United States, 35294
      • Data Coordination Center: University of Alabama at Birmingham
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Barrow Neurological Institute, Saint Joseph's Hospital and Medical Center, 350 W Thomas Rd
  • California
    • Los Angeles, California, United States, 90033
      • University of Southern California, Doheny Institute, 1450 San Pablo St
    • Orange, California, United States, 92868
      • University of California Irvine, 101 The City Drive S, Bldg. 22 C, Route 13
    • San Francisco, California, United States, 94114
      • California Pacific Medical Center, Castro St & Duboce Ave
  • Florida
    • Jacksonville, Florida, United States, 32209
      • University of Florida Jacksonville, Tower I, 8th Floor, 580 W. 8th ST.
    • Miami, Florida, United States, 33136
      • University of Miami, 1120 NW 14th Street, Suite 1300
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University, 201 Dowman Dr
    • Augusta, Georgia, United States, 30912
      • Augusta University, 1120 15th St
  • Indiana
    • Indianapolis, Indiana, United States, 46202-2859
      • Indiana University, Dept of Neurology, Regenstrief Health Center, 6th floor, 1050 Walnut St, Indiana University Medical Center
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • The University of Kansas Medical Center, 3901 Rainbow Blvd.
  • Massachusetts
    • Boston, Massachusetts, United States, 02115-6110
      • Brigham and Women's Hospital, 75 Francis Street, 5th Floor Tower
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Wayne State University School of Medicine, 4201 St Antoine, 8D UHC
    • Royal Oak, Michigan, United States, 48073
      • William Beaumont Hospital, 3601 W. Thirteen Mile Road, Royal Oak
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota, Department of Neurology, MMC 295, 420 Delaware St. S.E.,
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic Rochester, 200 First St. SW
  • Missouri
    • St. Louis, Missouri, United States, 63103-2097
      • St. Louis University, One North Grand St. Louis
  • New Jersey
    • New Brunswick, New Jersey, United States, 08901
      • Robert Wood Johnson University,
  • New York
    • New York, New York, United States, 10029
      • Mount Sinai Hospital,1 Gustave L. Levy Pl
    • Rochester, New York, United States, 14642
      • University of Rochester, 601 Elmwood Ave
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University, 200 Trent Dr
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Case Western Reserve University, University Hospitals of Cleveland, 1100 Euclid Avenue
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexmer Medical Center, Rm 461 Means Hall, The Ohio State University Medical Center,1654 Upham Dr.
  • Texas
    • Dallas, Texas, United States, 75390-8897
      • University of Texas Southwestern Medical Center, 5232 Harry Hines Blvd,
    • Galveston, Texas, United States, 77555-0539
      • University of Texas Medical Branch, 301 University Blvd
    • Houston, Texas, United States, 77030
      • Nerve and Muscle Center of Texas, 6624 Fannin St # 1670
    • San Antonio, Texas, United States, 78229-3900
      • University of Texas Health Science Center, Mail code 7883, 7703 Floyd Curl Drive
  • Vermont
    • Burlington, Vermont, United States, 05405
      • University of Vermont College of Medicine, Given Bldg C225, 89 Beaumont Avenue
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • University of Virginia, 1215 Lee St
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington, 1410 NE Campus Pkwy
  • West Virginia
    • Morgantown, West Virginia, United States, 26506
      • West Virginia University, Dept of Neurology, WVU Eye Institute, Neurology Suite, 1 Stadium Drive,
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Medical College of Wisconsin, 8701 Watertown Plank Road

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male and female MG patients age greater than 18 and less than 65 years
• Onset of generalized MG within the last 5 years
• Positive serum anti-acetylcholine receptor binding antibodies (muscle acetylcholine receptors, AchRAb =/> 1.00 nmol/L. AchRAb levels of 0.50-0.99 nmol/L will be acceptable if there is another confirmatory test for MG, including single-fiber electromyography (EMG), repetitive nerve stimulation, or unequivocal edrophonium testing.)
• MGFA class II-IV at entry, using the MG Foundation of America (MGFA) classification, while receiving optimal anti-cholinesterase treatment with or without oral prednisone

Exclusion Criteria:

• Ocular MG without generalized weakness (MGFA Class I) or minimal weakness that would not require the use of corticosteroids
• Myasthenic weakness requiring intubation (MGFA Class IV) in the prior month
• Immunosuppressive therapy other than corticosteroids in the preceding year
• Medically unfit for thymectomy
• Chest CT evidence of thymoma.
• Pregnancy or lactation; contraindications to the use of corticosteroids, unless postmenopausal or surgically sterile. Women considering becoming pregnant during the period of the study are to be excluded.
• A serious concurrent medical, neurological or psychiatric condition that would interfere with thymectomy or subsequent clinical assessments
• Current alternate day dose of prednisone > than 1.5 mg/kg or 100 mg or the equivalent daily doses (> 0.75 mg/kg or 50 mg).
• Participation in another experimental clinical trial
• History of alcohol or drug abuse within the 2 years prior to randomization.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Thymectomy plus prednisone; Procedure: Extended Transsternal Thymectomy plus prednisone treatment	Procedure: thymectomy plus prednisone; The thymectomy will be performed as soon as possible after randomization.; Other Names: Extended transsternal thymectomy plus prednisone
Placebo Comparator: Prednisone alone; Drug: prednisone alone protocol	Drug: prednisone alone; Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.; Other Names: prednisolone

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease. The time weighted average is a calculation that provides an integrated measure of the outcome over the time of followup. The denominator that was used to compute the time-weighted average for the Quantitative Myasthenia Gravis (QMG) score and the prednisone dose was the number of days from randomization to the last visit. Computations used the trapezoidal method where in the QMG score is multiplied by the number of days at this level from one visit to the next and added up over the entire followup experience and divided by the total number of days from randomization.	baseline, month 3, 4, 6 and every 3 months through 36 months
Time-weighted Average Alternate-day Prednisone Dose (mg) Measured Over 3 Years	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.	baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Prednisone Use at Enrollment	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.	baseline, month 3, 4, 6 and every 3 months through 36 months
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Sex	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.	baseline, month 3, 4, 6 and every 3 months through 36 months
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Age at Disease Onset	Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.	baseline, month 3, 4, 6 and every 3 months through 36 months
Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Prednisone Use at Enrollment	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.	baseline, month 3, 4, 6 and every 3 months through 36 months
Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Sex	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.	baseline, month 3, 4, 6 and every 3 months through 36 months
Subgroup Analyses of Time-weighted Average Average Alternate-day Prednisone Dose (mg) by Age at Disease Onset	Participants reported alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prednisone dosages had been weighted over the days of reporting period.	baseline, month 3, 4, 6 and every 3 months through 36 months
Number of Serious Adverse Events	Number of participant who experienced at least one serious adverse events over 3 years: Thymectomy plus prednisone n=25 (out of 66); Prednisone alone n=33 (out of 60)	baseline to 3 years
Number of Patients With at Least One Serious Adverse Events	Number of participant who experienced at least one serious adverse events over 3 years: Thymectomy plus prednisone n=25 (out of 66); Prednisone alone n=33 (out of 60)	baseline to 3 years
Classification of Serious Adverse Events		baseline to 3 years
Hospitalization for Exacerbation of Myasthenia Gravis		baseline to 2 years and baseline to 3 years
Cumulative Number of Hospital Days	Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)	baseline to 3 years
Reason for Hospitalization According to Medical Dictionary for Regulatory Activities Term	Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)	baseline to 3 years
Time-weighted Average Prescribed Alternate Day Prednisone Dose (mg)	Physicians reported prescribed alternate-day prednisone dose (mg) intake from baseline through withdrawn or completed 3 years follow up. The prescribed prednisone dosages had been weighted over the days of reporting period.	baseline-day 20, month 1,2, 3, 4, 6 and every 3 months through 36 months
Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 1: Penalized Using Maximum Dose Before Azathioprine)	For each participant who took azathioprine, we penalized them by taking the maximum dose of prednisone before azathioprine was added. We then applied the same method to compute the time-weighted alternative day prednisone dose from baseline, month 3, 4, 6 and every 3 months through 36 months.	baseline, month 3, 4, 6 and every 3 months through 36 months
Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 2: Penalized Using Dose at Time of Starting Azathioprine)	For each participant who took azathioprine, we penalized them by taking the prednisone dose at the time azathioprine commenced. We then applied the same method to compute the time-weighted alternative day prednisone dose from baseline, month 3, 4, 6 and every 3 months through 36 months.	baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months
Time-Weighted Average MG Activity of Daily Living (MG-ADL)	MG Activity of Daily Living total scores range from 0 to 24, with the lower scores indicating better daily living quality of life.	baseline, month 4, 6 and every 3 months through 36 months
Time-Weighted Average MG Activity of Daily Living (MG-ADL) at Month 12, 24, and 36	MG Activity of Daily Living total scores range from 0 to 24 by visit, with the lower scores indicating better daily living quality of life.	Month 12, 24, and 36
Azathioprine Use		baseline to 3 years
Plasma Exchange Use		baseline to 3 years
Intravenous Immunoglobulin Use		baseline to 3 years
Minimal Manifestation (MM) Status at Month 12, 24 and 36	Number of participants who were in minimal manifestation status at month 12, 24 and 36.	Month 12, 24 and 36
Cumulative Days in Hospital for Myasthenia Gravis Exacerbation	Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=17 (out of 60)	baseline to 2 years
Cumulative Days in Hospital for Myasthenia Gravis Exacerbation	Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=22 (out of 60)	baseline to 3 years
Short Form-36 Standardized Physical Component	Range from 0 to 100, the higher the physical component value, the better the mental health.	Month 0, Month 12, Month 24 and Month 36
Short Form-36 Standardized Mental Component	Range from 0 to 100, the higher the mental component value, the better the mental health.	Month 0, Month 12, Month 24 and Month 36
Treatment Associated Complications (TAC)	Treatment associated complications measured complications occurred by myasthenia gravis patients. Report number of participant with at least one complications by each visit.	Month 0, 1, 2, 3, 4 then every 3 months through Month 36
Treatment Associated Symptoms (TAS)	Treatment associated symptoms measured myasthenia gravis symptoms such as back pain and/or bruises. Report number of participant with at least one treatment associated symptoms by each visit.	Month 0, 1, 2, 3, 4 then every 3 months through Month 36

Collaborators and Investigators

• Sponsor: University of Alabama at Birmingham
• Collaborators: National Institute of Neurological Disorders and Stroke (NINDS)
• Investigators: Principal Investigator: Gary Cutter, PhD, University of Alabama at Birmingham School of Public Health, Department of Biostatistics; Principal Investigator: Gil Wolfe, MD, University of Buffalo, Jacobs School of Medicine and Biomedical Sciences; Principal Investigator: Henry Kaminski, MD, George Washington University School of Medicine and Health Sciences

Publications and helpful links

General Publications

• Aban IB, Wolfe GI, Cutter GR, Kaminski HJ, Jaretzki A 3rd, Minisman G, Conwit R, Newsom-Davis J; Mgtx Advisory Committee. The MGTX experience: challenges in planning and executing an international, multicenter clinical trial. J Neuroimmunol. 2008 Sep 15;201-202:80-4. doi: 10.1016/j.jneuroim.2008.05.031. Erratum In: J Neuroimmunol. 2009 Dec 10;217(1-2):103.
• Newsom-Davis J, Cutter G, Wolfe GI, Kaminski HJ, Jaretzki A 3rd, Minisman G, Aban I, Conwit R. Status of the thymectomy trial for nonthymomatous myasthenia gravis patients receiving prednisone. Ann N Y Acad Sci. 2008;1132:344-7. doi: 10.1196/annals.1405.014.
• Minisman G, Bhanushali M, Conwit R, Wolfe GI, Aban I, Kaminski HJ, Cutter G. Implementing clinical trials on an international platform: challenges and perspectives. J Neurol Sci. 2012 Feb 15;313(1-2):1-6. doi: 10.1016/j.jns.2011.10.004. Epub 2011 Nov 1.
• Kaminski HJ, Kusner LL, Wolfe GI, Aban I, Minisman G, Conwit R, Cutter G. Biomarker development for myasthenia gravis. Ann N Y Acad Sci. 2012 Dec;1275(1):101-6. doi: 10.1111/j.1749-6632.2012.06787.x.
• Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Strobel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BR, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Garcia Ramos GS, Verschuuren JJ, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Odenkirchen J, Sonett JR, Jaretzki A 3rd, Newsom-Davis J, Cutter GR; MGTX Study Group. Randomized Trial of Thymectomy in Myasthenia Gravis. N Engl J Med. 2016 Aug 11;375(6):511-22. doi: 10.1056/NEJMoa1602489. Erratum In: N Engl J Med. 2017 May 25;376(21):2097. [Dosage error in article text].
• Lee I, Kuo HC, Aban IB, Cutter GR, McPherson T, Kaminski HJ, Sussman J, Strobel P, Oger J, Cea G, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJG, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Minisman G, Sonett JR, Wolfe GI; MGTX study group. Minimal manifestation status and prednisone withdrawal in the MGTX trial. Neurology. 2020 Aug 11;95(6):e755-e766. doi: 10.1212/WNL.0000000000010031. Epub 2020 Jul 1.
• Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Strobel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJGM, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Silvestri NJ, Conwit R, Sonett JR, Jaretzki A 3rd, Newsom-Davis J, Cutter GR; MGTX Study Group. Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial. Lancet Neurol. 2019 Mar;18(3):259-268. doi: 10.1016/S1474-4422(18)30392-2. Epub 2019 Jan 25.

Study record dates

Study Major Dates

• Study Start: June 1, 2006
• Primary Completion (Actual): November 1, 2015
• Study Completion (Actual): December 1, 2015

Study Registration Dates

• First Submitted: February 21, 2006
• First Submitted That Met QC Criteria: February 21, 2006
• First Posted (Estimate): February 22, 2006

Study Record Updates

• Last Update Posted (Actual): May 23, 2017
• Last Update Submitted That Met QC Criteria: April 12, 2017
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Keywords: MG; corticosteroid; myasthenia gravis; prednisone; thymoma; thymectomy; thymus; extended transsternal thymectomy; ETTX
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis; Physiological Effects of Drugs; Anti-Inflammatory Agents; Antineoplastic Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Prednisolone; Prednisone
• Other Study ID Numbers: R01NS050733; CRC (NINDS); 1U01NS042685-01A2 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES