Back to resultsThymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy
Primary Purpose
Myasthenia Gravis
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
thymectomy plus prednisone
prednisone alone
Sponsored by
About this trial
This is an interventional treatment trial for Myasthenia Gravis focused on measuring myasthenia gravis, thymectomy, prednisone, corticosteroid, extended transsternal thymectomy, ETTX, MG, thymus, thymoma
Eligibility Criteria
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.
Sites / Locations
- University of Alabama at Birmingham, Department of Neurology, Sparks Center, Suite 350, 1720 7th Avenue South
- Data Coordination Center: University of Alabama at Birmingham
- Barrow Neurological Institute, Saint Joseph's Hospital and Medical Center, 350 W Thomas Rd
- University of Southern California, Doheny Institute, 1450 San Pablo St
- University of California Irvine, 101 The City Drive S, Bldg. 22 C, Route 13
- California Pacific Medical Center, Castro St & Duboce Ave
- University of Florida Jacksonville, Tower I, 8th Floor, 580 W. 8th ST.
- University of Miami, 1120 NW 14th Street, Suite 1300
- Emory University, 201 Dowman Dr
- Augusta University, 1120 15th St
- Indiana University, Dept of Neurology, Regenstrief Health Center, 6th floor, 1050 Walnut St, Indiana University Medical Center
- The University of Kansas Medical Center, 3901 Rainbow Blvd.
- Brigham and Women's Hospital, 75 Francis Street, 5th Floor Tower
- Wayne State University School of Medicine, 4201 St Antoine, 8D UHC
- William Beaumont Hospital, 3601 W. Thirteen Mile Road, Royal Oak
- University of Minnesota, Department of Neurology, MMC 295, 420 Delaware St. S.E.,
- Mayo Clinic Rochester, 200 First St. SW
- St. Louis University, One North Grand St. Louis
- Robert Wood Johnson University,
- Mount Sinai Hospital,1 Gustave L. Levy Pl
- University of Rochester, 601 Elmwood Ave
- Duke University, 200 Trent Dr
- Case Western Reserve University, University Hospitals of Cleveland, 1100 Euclid Avenue
- The Ohio State University Wexmer Medical Center, Rm 461 Means Hall, The Ohio State University Medical Center,1654 Upham Dr.
- University of Texas Southwestern Medical Center, 5232 Harry Hines Blvd,
- University of Texas Medical Branch, 301 University Blvd
- Nerve and Muscle Center of Texas, 6624 Fannin St # 1670
- University of Texas Health Science Center, Mail code 7883, 7703 Floyd Curl Drive
- University of Vermont College of Medicine, Given Bldg C225, 89 Beaumont Avenue
- University of Virginia, 1215 Lee St
- University of Washington, 1410 NE Campus Pkwy
- West Virginia University, Dept of Neurology, WVU Eye Institute, Neurology Suite, 1 Stadium Drive,
- Medical College of Wisconsin, 8701 Watertown Plank Road
- University of Buenis, Centro de Asistencia Docencia e Investigacion en Miastenia (CADIMI) Av. Forest 1146 - Ciudad Autonoma de Buenos Aires
- University of Sydney, Royal Prince Alfred Hospital and The University of Sydney
- University of Melbourne, Melbourne, The Royal Melbourne Hospital, Dept of Neurology, Royal Melbourne Hospital
- Hospital de Base do Distrito Federal
- Universidade Federal do Parana
- Federal University of Rio De Janeiro
- University of Calgary, Heritage Medical Research Clinic Room 1132 3330 Hospital Dr NW
- University of British Columbia
- University of Ottawa, The Ottawa Hospital General Campus, Division of Neurology, 501 Smyth Rd. Box 601
- McGill University Health Center
- Hospital Del Salvador, Departamento de Ciencias Neurológicas, Universidad de Chile, Salvador 95 Of 416, Providencia
- University of Heidelberg, Seminarstraße 2
- University of Regensburg, Dept. of Neurology, Universitätsstr. 84, D
- University of Düsseldorf
- Johannes-Gutenberg University, Klinikum der Johannes Gutenberg-Universität, Klinik und Poliklinik für Neurologie, Langenbeckstr
- University of Münster, Schlossplatz 2
- University of Tübingen
- National Neurological Institute "Carlo Besta", Myopathology and Immunology Unit, Dept of Neurology IV, Natl. Neurolog Inst. "C. Besta", Via Celoria, 11,
- University of Rome "Sapienza"
- Catholic University, Universita Cattolica del Sacro Cuore, Largo Agostino Gemelli 8, e
- University of Torino
- Kanazawa University, Department of Neurology, Kanazawa University Hospital, 13-1 Takaramachi
- Nagasaki University, First Department of Internal Medicine,Graduate School of Biomedical Sciences,1-7-1,Sakamoto
- Instituto Nacional de la Nutrición
- Leiden University
- Medical University of Warsaw
- Institute of Tuberculosis and Lung Disease
- Porto University, Serviço de Neurologia,Hospital Geral de Santo António, Largo Prof Abel Salazar
- University of Cape Town, Division of Neurology E8-74, Groote Schuur Hospital,Observatory
- H. Sant Pau, Universitat Autònoma de Barcelona, Neurology Department, Hospital Sta Creu i Sant Pau, C/Mas Casanovas no 90 4o pis 4o modul.
- Fu-Jen Catholic University, No. 510, Zhongzheng Rd., Xinzhuang Dist
- Ramathibodi Hospital, Mahidol University
- Queen Elizabeth University Hospital, Glasgow
- Walton Centre for Neurology and Neurosurgery, Liverpool Heart and Chest Hospital, Liverpool. The Walton Centre for Neurology and Neurosurgery, Lower Lane, Fazakerley
- University of Manchester, Oxford Road
- University of Oxford, Dept of Clinical Neurology, University of Oxford, Radcliffe Infirmary
- University of Sheffield, Western Bank
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
Thymectomy plus prednisone
Prednisone alone
Arm Description
Procedure: Extended Transsternal Thymectomy plus prednisone treatment
Drug: prednisone alone protocol
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
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.
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.
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.
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.
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.
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.
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)
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)
Classification of Serious Adverse Events
Hospitalization for Exacerbation of Myasthenia Gravis
Cumulative Number of Hospital Days
Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)
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)
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.
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.
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.
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.
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.
Azathioprine Use
Plasma Exchange Use
Intravenous Immunoglobulin Use
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.
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)
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)
Short Form-36 Standardized Physical Component
Range from 0 to 100, the higher the physical component value, the better the mental health.
Short Form-36 Standardized Mental Component
Range from 0 to 100, the higher the mental component value, the better the mental health.
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.
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.
Full Information
NCT ID
NCT00294658
First Posted
February 21, 2006
Last Updated
April 12, 2017
Sponsor
University of Alabama at Birmingham
Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)
1. Study Identification
Unique Protocol Identification Number
NCT00294658
Brief Title
Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy
Official Title
A Multi-Center, Single-Blind, Randomized Study Comparing Thymectomy to No Thymectomy in Non-Thymomatous Myasthenia Gravis (MG) Patients Receiving Prednisone
Study Type
Interventional
2. Study Status
Record Verification Date
April 2017
Overall Recruitment Status
Completed
Study Start Date
June 2006 (undefined)
Primary Completion Date
November 2015 (Actual)
Study Completion Date
December 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Alabama at Birmingham
Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myasthenia Gravis
Keywords
myasthenia gravis, thymectomy, prednisone, corticosteroid, extended transsternal thymectomy, ETTX, MG, thymus, thymoma
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
Outcomes Assessor
Allocation
Randomized
Enrollment
126 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Thymectomy plus prednisone
Arm Type
Active Comparator
Arm Description
Procedure: Extended Transsternal Thymectomy plus prednisone treatment
Arm Title
Prednisone alone
Arm Type
Placebo Comparator
Arm Description
Drug: prednisone alone protocol
Intervention Type
Procedure
Intervention Name(s)
thymectomy plus prednisone
Other Intervention Name(s)
Extended transsternal thymectomy plus prednisone
Intervention Description
The thymectomy will be performed as soon as possible after randomization.
Intervention Type
Drug
Intervention Name(s)
prednisone alone
Other Intervention Name(s)
prednisolone
Intervention Description
Prednisone regimen will be every other day, starting at 10mg. The dose will increase by 10mg every 2 days to a target dose.
Primary Outcome Measure Information:
Title
Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years
Description
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.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Time-weighted Average Alternate-day Prednisone Dose (mg) Measured Over 3 Years
Description
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.
Time Frame
baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months
Secondary Outcome Measure Information:
Title
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Prednisone Use at Enrollment
Description
Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Sex
Description
Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Age at Disease Onset
Description
Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Prednisone Use at Enrollment
Description
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.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Sex
Description
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.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Subgroup Analyses of Time-weighted Average Average Alternate-day Prednisone Dose (mg) by Age at Disease Onset
Description
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.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Number of Serious Adverse Events
Description
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)
Time Frame
baseline to 3 years
Title
Number of Patients With at Least One Serious Adverse Events
Description
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)
Time Frame
baseline to 3 years
Title
Classification of Serious Adverse Events
Time Frame
baseline to 3 years
Title
Hospitalization for Exacerbation of Myasthenia Gravis
Time Frame
baseline to 2 years and baseline to 3 years
Title
Cumulative Number of Hospital Days
Description
Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)
Time Frame
baseline to 3 years
Title
Reason for Hospitalization According to Medical Dictionary for Regulatory Activities Term
Description
Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)
Time Frame
baseline to 3 years
Title
Time-weighted Average Prescribed Alternate Day Prednisone Dose (mg)
Description
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.
Time Frame
baseline-day 20, month 1,2, 3, 4, 6 and every 3 months through 36 months
Title
Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 1: Penalized Using Maximum Dose Before Azathioprine)
Description
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.
Time Frame
baseline, month 3, 4, 6 and every 3 months through 36 months
Title
Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 2: Penalized Using Dose at Time of Starting Azathioprine)
Description
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.
Time Frame
baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months
Title
Time-Weighted Average MG Activity of Daily Living (MG-ADL)
Description
MG Activity of Daily Living total scores range from 0 to 24, with the lower scores indicating better daily living quality of life.
Time Frame
baseline, month 4, 6 and every 3 months through 36 months
Title
Time-Weighted Average MG Activity of Daily Living (MG-ADL) at Month 12, 24, and 36
Description
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.
Time Frame
Month 12, 24, and 36
Title
Azathioprine Use
Time Frame
baseline to 3 years
Title
Plasma Exchange Use
Time Frame
baseline to 3 years
Title
Intravenous Immunoglobulin Use
Time Frame
baseline to 3 years
Title
Minimal Manifestation (MM) Status at Month 12, 24 and 36
Description
Number of participants who were in minimal manifestation status at month 12, 24 and 36.
Time Frame
Month 12, 24 and 36
Title
Cumulative Days in Hospital for Myasthenia Gravis Exacerbation
Description
Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=17 (out of 60)
Time Frame
baseline to 2 years
Title
Cumulative Days in Hospital for Myasthenia Gravis Exacerbation
Description
Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=22 (out of 60)
Time Frame
baseline to 3 years
Title
Short Form-36 Standardized Physical Component
Description
Range from 0 to 100, the higher the physical component value, the better the mental health.
Time Frame
Month 0, Month 12, Month 24 and Month 36
Title
Short Form-36 Standardized Mental Component
Description
Range from 0 to 100, the higher the mental component value, the better the mental health.
Time Frame
Month 0, Month 12, Month 24 and Month 36
Title
Treatment Associated Complications (TAC)
Description
Treatment associated complications measured complications occurred by myasthenia gravis patients. Report number of participant with at least one complications by each visit.
Time Frame
Month 0, 1, 2, 3, 4 then every 3 months through Month 36
Title
Treatment Associated Symptoms (TAS)
Description
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.
Time Frame
Month 0, 1, 2, 3, 4 then every 3 months through Month 36
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gary Cutter, PhD
Organizational Affiliation
University of Alabama at Birmingham School of Public Health, Department of Biostatistics
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Gil Wolfe, MD
Organizational Affiliation
University of Buffalo, Jacobs School of Medicine and Biomedical Sciences
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Henry Kaminski, MD
Organizational Affiliation
George Washington University School of Medicine and Health Sciences
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Alabama at Birmingham, Department of Neurology, Sparks Center, Suite 350, 1720 7th Avenue South
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Data Coordination Center: University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Barrow Neurological Institute, Saint Joseph's Hospital and Medical Center, 350 W Thomas Rd
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
University of Southern California, Doheny Institute, 1450 San Pablo St
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
University of California Irvine, 101 The City Drive S, Bldg. 22 C, Route 13
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
California Pacific Medical Center, Castro St & Duboce Ave
City
San Francisco
State/Province
California
ZIP/Postal Code
94114
Country
United States
Facility Name
University of Florida Jacksonville, Tower I, 8th Floor, 580 W. 8th ST.
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32209
Country
United States
Facility Name
University of Miami, 1120 NW 14th Street, Suite 1300
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Emory University, 201 Dowman Dr
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Augusta University, 1120 15th St
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Indiana University, Dept of Neurology, Regenstrief Health Center, 6th floor, 1050 Walnut St, Indiana University Medical Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202-2859
Country
United States
Facility Name
The University of Kansas Medical Center, 3901 Rainbow Blvd.
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Brigham and Women's Hospital, 75 Francis Street, 5th Floor Tower
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115-6110
Country
United States
Facility Name
Wayne State University School of Medicine, 4201 St Antoine, 8D UHC
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
William Beaumont Hospital, 3601 W. Thirteen Mile Road, Royal Oak
City
Royal Oak
State/Province
Michigan
ZIP/Postal Code
48073
Country
United States
Facility Name
University of Minnesota, Department of Neurology, MMC 295, 420 Delaware St. S.E.,
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Mayo Clinic Rochester, 200 First St. SW
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
St. Louis University, One North Grand St. Louis
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63103-2097
Country
United States
Facility Name
Robert Wood Johnson University,
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
Mount Sinai Hospital,1 Gustave L. Levy Pl
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
University of Rochester, 601 Elmwood Ave
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Duke University, 200 Trent Dr
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Case Western Reserve University, University Hospitals of Cleveland, 1100 Euclid Avenue
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
The Ohio State University Wexmer Medical Center, Rm 461 Means Hall, The Ohio State University Medical Center,1654 Upham Dr.
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
University of Texas Southwestern Medical Center, 5232 Harry Hines Blvd,
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-8897
Country
United States
Facility Name
University of Texas Medical Branch, 301 University Blvd
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555-0539
Country
United States
Facility Name
Nerve and Muscle Center of Texas, 6624 Fannin St # 1670
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Texas Health Science Center, Mail code 7883, 7703 Floyd Curl Drive
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229-3900
Country
United States
Facility Name
University of Vermont College of Medicine, Given Bldg C225, 89 Beaumont Avenue
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05405
Country
United States
Facility Name
University of Virginia, 1215 Lee St
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
University of Washington, 1410 NE Campus Pkwy
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
West Virginia University, Dept of Neurology, WVU Eye Institute, Neurology Suite, 1 Stadium Drive,
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
Medical College of Wisconsin, 8701 Watertown Plank Road
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
University of Buenis, Centro de Asistencia Docencia e Investigacion en Miastenia (CADIMI) Av. Forest 1146 - Ciudad Autonoma de Buenos Aires
City
Buenos Aires
Country
Argentina
Facility Name
University of Sydney, Royal Prince Alfred Hospital and The University of Sydney
City
Sydney
Country
Australia
Facility Name
University of Melbourne, Melbourne, The Royal Melbourne Hospital, Dept of Neurology, Royal Melbourne Hospital
City
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
Hospital de Base do Distrito Federal
City
Brasilia
ZIP/Postal Code
CEP 71640 255
Country
Brazil
Facility Name
Universidade Federal do Parana
City
Curitiba
ZIP/Postal Code
80060-900
Country
Brazil
Facility Name
Federal University of Rio De Janeiro
City
Rio De Janeiro
ZIP/Postal Code
CEP 20520-053
Country
Brazil
Facility Name
University of Calgary, Heritage Medical Research Clinic Room 1132 3330 Hospital Dr NW
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 2T9
Country
Canada
Facility Name
University of British Columbia
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6T 2B5
Country
Canada
Facility Name
University of Ottawa, The Ottawa Hospital General Campus, Division of Neurology, 501 Smyth Rd. Box 601
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L6
Country
Canada
Facility Name
McGill University Health Center
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3G 1A4
Country
Canada
Facility Name
Hospital Del Salvador, Departamento de Ciencias Neurológicas, Universidad de Chile, Salvador 95 Of 416, Providencia
City
Santiago
Country
Chile
Facility Name
University of Heidelberg, Seminarstraße 2
City
Mannheim
State/Province
Baden-Württemberg
ZIP/Postal Code
69117
Country
Germany
Facility Name
University of Regensburg, Dept. of Neurology, Universitätsstr. 84, D
City
Regensburg
State/Province
Bavaria
ZIP/Postal Code
93043
Country
Germany
Facility Name
University of Düsseldorf
City
Düsseldorf
State/Province
North Rhine-Westphalia
ZIP/Postal Code
D-40225
Country
Germany
Facility Name
Johannes-Gutenberg University, Klinikum der Johannes Gutenberg-Universität, Klinik und Poliklinik für Neurologie, Langenbeckstr
City
Mainz
State/Province
Rhineland-Palatinate
ZIP/Postal Code
55101
Country
Germany
Facility Name
University of Münster, Schlossplatz 2
City
Münster
ZIP/Postal Code
48149
Country
Germany
Facility Name
University of Tübingen
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
National Neurological Institute "Carlo Besta", Myopathology and Immunology Unit, Dept of Neurology IV, Natl. Neurolog Inst. "C. Besta", Via Celoria, 11,
City
Milan
ZIP/Postal Code
20133
Country
Italy
Facility Name
University of Rome "Sapienza"
City
Rome
ZIP/Postal Code
00189
Country
Italy
Facility Name
Catholic University, Universita Cattolica del Sacro Cuore, Largo Agostino Gemelli 8, e
City
Rome
Country
Italy
Facility Name
University of Torino
City
Torino
Country
Italy
Facility Name
Kanazawa University, Department of Neurology, Kanazawa University Hospital, 13-1 Takaramachi
City
Kanazawa
State/Province
Ishikawa
ZIP/Postal Code
920-8641
Country
Japan
Facility Name
Nagasaki University, First Department of Internal Medicine,Graduate School of Biomedical Sciences,1-7-1,Sakamoto
City
Nagasaki
State/Province
Kyushu
ZIP/Postal Code
852-8501
Country
Japan
Facility Name
Instituto Nacional de la Nutrición
City
Mexico
ZIP/Postal Code
14000
Country
Mexico
Facility Name
Leiden University
City
Leiden
Country
Netherlands
Facility Name
Medical University of Warsaw
City
Warsaw
State/Province
Województwo
ZIP/Postal Code
02 097
Country
Poland
Facility Name
Institute of Tuberculosis and Lung Disease
City
Warsaw
State/Province
Województwo
Country
Poland
Facility Name
Porto University, Serviço de Neurologia,Hospital Geral de Santo António, Largo Prof Abel Salazar
City
Porto
ZIP/Postal Code
4099-001
Country
Portugal
Facility Name
University of Cape Town, Division of Neurology E8-74, Groote Schuur Hospital,Observatory
City
Cape Town
Country
South Africa
Facility Name
H. Sant Pau, Universitat Autònoma de Barcelona, Neurology Department, Hospital Sta Creu i Sant Pau, C/Mas Casanovas no 90 4o pis 4o modul.
City
Barcelona
ZIP/Postal Code
08025
Country
Spain
Facility Name
Fu-Jen Catholic University, No. 510, Zhongzheng Rd., Xinzhuang Dist
City
New Taipei
ZIP/Postal Code
24205
Country
Taiwan
Facility Name
Ramathibodi Hospital, Mahidol University
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Facility Name
Queen Elizabeth University Hospital, Glasgow
City
Glasgow
ZIP/Postal Code
G51 4TF
Country
United Kingdom
Facility Name
Walton Centre for Neurology and Neurosurgery, Liverpool Heart and Chest Hospital, Liverpool. The Walton Centre for Neurology and Neurosurgery, Lower Lane, Fazakerley
City
Liverpool
ZIP/Postal Code
L9 7LJ
Country
United Kingdom
Facility Name
University of Manchester, Oxford Road
City
Manchester
ZIP/Postal Code
M13 9PL
Country
United Kingdom
Facility Name
University of Oxford, Dept of Clinical Neurology, University of Oxford, Radcliffe Infirmary
City
Oxford
ZIP/Postal Code
OX2 6HE
Country
United Kingdom
Facility Name
University of Sheffield, Western Bank
City
Sheffield
ZIP/Postal Code
S10 2TN
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
Citations:
PubMed Identifier
18675464
Citation
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.
Results Reference
result
PubMed Identifier
18567886
Citation
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.
Results Reference
result
PubMed Identifier
22047648
Citation
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.
Results Reference
result
PubMed Identifier
23278584
Citation
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.
Results Reference
result
PubMed Identifier
27509100
Citation
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].
Results Reference
result
PubMed Identifier
32611638
Citation
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.
Results Reference
derived
PubMed Identifier
30692052
Citation
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.
Results Reference
derived
Learn more about this trial
Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy
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