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Myasthenia GravisPhase 3NCT00294658

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

A Phase 3 study testing thymectomy plus prednisone and prednisone alone in people with Myasthenia Gravis, run by University of Alabama at Birmingham at 70 sites in 18 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Myasthenia Gravis
What is being tested?
thymectomy plus prednisone and prednisone alone
Type of treatment
Surgery or procedure
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
126 participants
Where
70 sitesin 18 countries
Study dates
Jun 2006to Dec 2015
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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.
Show all 10Show fewer
  • 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.

Trial locations and contacts

70 sites in 18 countries.

70 sites in 18 countries

United States33 sites

  • Emory University, 201 Dowman Dr

    Atlanta, Georgia, 30322

  • Augusta University, 1120 15th St

    Augusta, Georgia, 30912

  • University of Alabama at Birmingham, Department of Neurology, Sparks Center, Suite 350, 1720 7th Avenue South

    Birmingham, Alabama, 35233

  • Data Coordination Center: University of Alabama at Birmingham

    Birmingham, Alabama, 35294

  • Brigham and Women's Hospital, 75 Francis Street, 5th Floor Tower

    Boston, Massachusetts, 02115-6110

  • University of Vermont College of Medicine, Given Bldg C225, 89 Beaumont Avenue

    Burlington, Vermont, 05405

  • University of Virginia, 1215 Lee St

    Charlottesville, Virginia, 22908

  • Case Western Reserve University, University Hospitals of Cleveland, 1100 Euclid Avenue

    Cleveland, Ohio, 44106

  • The Ohio State University Wexmer Medical Center, Rm 461 Means Hall, The Ohio State University Medical Center,1654 Upham Dr.

    Columbus, Ohio, 43210

  • University of Texas Southwestern Medical Center, 5232 Harry Hines Blvd,

    Dallas, Texas, 75390-8897

  • Wayne State University School of Medicine, 4201 St Antoine, 8D UHC

    Detroit, Michigan, 48201

  • Duke University, 200 Trent Dr

    Durham, North Carolina, 27710

  • University of Texas Medical Branch, 301 University Blvd

    Galveston, Texas, 77555-0539

  • Nerve and Muscle Center of Texas, 6624 Fannin St # 1670

    Houston, Texas, 77030

  • Indiana University, Dept of Neurology, Regenstrief Health Center, 6th floor, 1050 Walnut St, Indiana University Medical Center

    Indianapolis, Indiana, 46202-2859

  • University of Florida Jacksonville, Tower I, 8th Floor, 580 W. 8th ST.

    Jacksonville, Florida, 32209

  • The University of Kansas Medical Center, 3901 Rainbow Blvd.

    Kansas City, Kansas, 66160

  • University of Southern California, Doheny Institute, 1450 San Pablo St

    Los Angeles, California, 90033

  • University of Miami, 1120 NW 14th Street, Suite 1300

    Miami, Florida, 33136

  • Medical College of Wisconsin, 8701 Watertown Plank Road

    Milwaukee, Wisconsin, 53226

  • University of Minnesota, Department of Neurology, MMC 295, 420 Delaware St. S.E.,

    Minneapolis, Minnesota, 55455

  • West Virginia University, Dept of Neurology, WVU Eye Institute, Neurology Suite, 1 Stadium Drive,

    Morgantown, West Virginia, 26506

  • Robert Wood Johnson University,

    New Brunswick, New Jersey, 08901

  • Mount Sinai Hospital,1 Gustave L. Levy Pl

    New York, New York, 10029

  • University of California Irvine, 101 The City Drive S, Bldg. 22 C, Route 13

    Orange, California, 92868

  • Barrow Neurological Institute, Saint Joseph's Hospital and Medical Center, 350 W Thomas Rd

    Phoenix, Arizona, 85016

  • Mayo Clinic Rochester, 200 First St. SW

    Rochester, Minnesota, 55905

  • University of Rochester, 601 Elmwood Ave

    Rochester, New York, 14642

  • William Beaumont Hospital, 3601 W. Thirteen Mile Road, Royal Oak

    Royal Oak, Michigan, 48073

  • University of Texas Health Science Center, Mail code 7883, 7703 Floyd Curl Drive

    San Antonio, Texas, 78229-3900

  • California Pacific Medical Center, Castro St & Duboce Ave

    San Francisco, California, 94114

  • University of Washington, 1410 NE Campus Pkwy

    Seattle, Washington, 98195

  • St. Louis University, One North Grand St. Louis

    St Louis, Missouri, 63103-2097

Other trials in United States

Germany6 sites

  • University of Düsseldorf

    Düsseldorf, North Rhine-Westphalia, D-40225

  • Johannes-Gutenberg University, Klinikum der Johannes Gutenberg-Universität, Klinik und Poliklinik für Neurologie, Langenbeckstr

    Mainz, Rhineland-Palatinate, 55101

  • University of Heidelberg, Seminarstraße 2

    Mannheim, Baden-Wurttemberg, 69117

  • University of Münster, Schlossplatz 2

    Münster, 48149

  • University of Regensburg, Dept. of Neurology, Universitätsstr. 84, D

    Regensburg, Bavaria, 93043

  • University of Tübingen

    Tübingen, 72076

Other trials in Germany

United Kingdom5 sites

  • Queen Elizabeth University Hospital, Glasgow

    Glasgow, G51 4TF

  • Walton Centre for Neurology and Neurosurgery, Liverpool Heart and Chest Hospital, Liverpool. The Walton Centre for Neurology and Neurosurgery, Lower Lane, Fazakerley

    Liverpool, L9 7LJ

  • University of Manchester, Oxford Road

    Manchester, M13 9PL

  • University of Oxford, Dept of Clinical Neurology, University of Oxford, Radcliffe Infirmary

    Oxford, OX2 6HE

  • University of Sheffield, Western Bank

    Sheffield, S10 2TN

Other trials in United Kingdom

Canada4 sites

  • University of Calgary, Heritage Medical Research Clinic Room 1132 3330 Hospital Dr NW

    Calgary, Alberta, T2N 2T9

  • McGill University Health Center

    Montreal, Quebec, H3G 1A4

  • University of Ottawa, The Ottawa Hospital General Campus, Division of Neurology, 501 Smyth Rd. Box 601

    Ottawa, Ontario, K1H 8L6

  • University of British Columbia

    Vancouver, British Columbia, V6T 2B5

Other trials in Canada

Italy4 sites

  • National Neurological Institute "Carlo Besta", Myopathology and Immunology Unit, Dept of Neurology IV, Natl. Neurolog Inst. "C. Besta", Via Celoria, 11,

    Milan, 20133

  • University of Rome "Sapienza"

    Rome, 00189

  • Catholic University, Universita Cattolica del Sacro Cuore, Largo Agostino Gemelli 8, e

    Rome

  • University of Torino

    Torino

Other trials in Italy

Brazil3 sites

  • Hospital de Base do Distrito Federal

    Brasília, CEP 71640 255

  • Universidade Federal do Parana

    Curitiba, 80060-900

  • Federal University of Rio De Janeiro

    Rio de Janeiro, CEP 20520-053

Other trials in Brazil

Australia2 sites

  • University of Sydney, Royal Prince Alfred Hospital and The University of Sydney

    Sydney

  • University of Melbourne, Melbourne, The Royal Melbourne Hospital, Dept of Neurology, Royal Melbourne Hospital

    Victoria, 3050

Other trials in Australia

Japan2 sites

  • Kanazawa University, Department of Neurology, Kanazawa University Hospital, 13-1 Takaramachi

    Kanazawa, Ishikawa-ken, 920-8641

  • Nagasaki University, First Department of Internal Medicine,Graduate School of Biomedical Sciences,1-7-1,Sakamoto

    Nagasaki, Kyushu, 852-8501

Other trials in Japan

Poland2 sites

  • Medical University of Warsaw

    Warsaw, Województwo, 02 097

  • Institute of Tuberculosis and Lung Disease

    Warsaw, Województwo

Other trials in Poland

Argentina1 site

  • University of Buenis, Centro de Asistencia Docencia e Investigacion en Miastenia (CADIMI) Av. Forest 1146 - Ciudad Autonoma de Buenos Aires

    Buenos Aires

Other trials in Argentina

Chile1 site

  • Hospital Del Salvador, Departamento de Ciencias Neurológicas, Universidad de Chile, Salvador 95 Of 416, Providencia

    Santiago

Other trials in Chile

Mexico1 site

  • Instituto Nacional de la Nutrición

    México, 14000

Other trials in Mexico

Netherlands1 site

  • Leiden University

    Leiden

Other trials in Netherlands

Portugal1 site

  • Porto University, Serviço de Neurologia,Hospital Geral de Santo António, Largo Prof Abel Salazar

    Porto, 4099-001

Other trials in Portugal

South Africa1 site

  • University of Cape Town, Division of Neurology E8-74, Groote Schuur Hospital,Observatory

    Cape Town

Other trials in South Africa

Spain1 site

  • H. Sant Pau, Universitat Autònoma de Barcelona, Neurology Department, Hospital Sta Creu i Sant Pau, C/Mas Casanovas no 90 4o pis 4o modul.

    Barcelona, 08025

Other trials in Spain

Taiwan1 site

  • Fu-Jen Catholic University, No. 510, Zhongzheng Rd., Xinzhuang Dist

    New Taipei City, 24205

Other trials in Taiwan

Thailand1 site

  • Ramathibodi Hospital, Mahidol University

    Bangkok, 10400

Other trials in Thailand

About this study

Official title: 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.

Read the 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.

Being tested
thymectomy plus prednisone, prednisone alone
Keywords
myasthenia gravis, thymectomy, prednisone, corticosteroid, extended transsternal thymectomy, ETTX, MG, thymus, thymoma
Medical subject headings
Myasthenia Gravis, Thymoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • thymectomy plus prednisoneProcedure or surgery

    Also called Extended transsternal thymectomy plus prednisone

    The thymectomy will be performed as soon as possible after randomization.

  • prednisone aloneDrug

    Also called prednisolone

    Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.

Study groups in NCT00294658
Study groupRoleWhat they receive
Thymectomy plus prednisoneActive comparatorProcedure: Extended Transsternal Thymectomy plus prednisone treatment
Receives: Procedure: thymectomy plus prednisone
Prednisone alonePlacebo comparatorDrug: prednisone alone protocol
Receives: Drug: prednisone alone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  2. 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.

    Measured: baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months

Secondary outcomes (27)

  1. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  2. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  3. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  4. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  5. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  6. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  7. 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)

    Measured: baseline to 3 years

  8. 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)

    Measured: baseline to 3 years

  9. Classification of Serious Adverse Events

    Measured: baseline to 3 years

  10. Hospitalization for Exacerbation of Myasthenia Gravis

    Measured: baseline to 2 years and baseline to 3 years

  11. Cumulative Number of Hospital Days

    Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)

    Measured: baseline to 3 years

  12. 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)

    Measured: baseline to 3 years

  13. 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.

    Measured: baseline-day 20, month 1,2, 3, 4, 6 and every 3 months through 36 months

  14. 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.

    Measured: baseline, month 3, 4, 6 and every 3 months through 36 months

  15. 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.

    Measured: baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months

  16. 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.

    Measured: baseline, month 4, 6 and every 3 months through 36 months

  17. 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.

    Measured: Month 12, 24, and 36

  18. Azathioprine Use

    Measured: baseline to 3 years

  19. Plasma Exchange Use

    Measured: baseline to 3 years

  20. Intravenous Immunoglobulin Use

    Measured: baseline to 3 years

  21. 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.

    Measured: Month 12, 24 and 36

  22. 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)

    Measured: baseline to 2 years

  23. 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)

    Measured: baseline to 3 years

  24. Short Form-36 Standardized Physical Component

    Range from 0 to 100, the higher the physical component value, the better the mental health.

    Measured: Month 0, Month 12, Month 24 and Month 36

  25. Short Form-36 Standardized Mental Component

    Range from 0 to 100, the higher the mental component value, the better the mental health.

    Measured: Month 0, Month 12, Month 24 and Month 36

  26. 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.

    Measured: Month 0, 1, 2, 3, 4 then every 3 months through Month 36

  27. 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.

    Measured: Month 0, 1, 2, 3, 4 then every 3 months through Month 36

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Feb 2006 (estimated)
  2. Study startJun 2006
  3. Primary completionNov 2015
  4. Study completionDec 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Outcomes assessor
Enrolment
126 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00294658
Sponsor's protocol ID
R01NS050733
Other IDs
1U01NS042685-01A2, CRC
Status last verified
Apr 2017
First submitted
21 Feb 2006
Last updated
23 May 2017
Data monitoring committee
Yes
Individual data sharing
Yes

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.

    PubMed 18675464

  • 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.

    PubMed 18567886

  • 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.

    PubMed 22047648

  • 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.

    PubMed 23278584

  • 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.

    PubMed 27509100

  • 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.

    PubMed 32611638

  • 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.

    PubMed 30692052

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing thymectomy plus prednisone and prednisone alone in people with Myasthenia Gravis, run by University of Alabama at Birmingham at 70 sites in 18 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 May 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 70 sites in United States, Germany, United Kingdom, Canada, Italy and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Jan 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years (time frame: baseline, month 3, 4, 6 and every 3 months through 36 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00294658, which also lists 7 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2017; AzTrials last refreshed its copy on 8 Nov 2026.

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