Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT00294658
Registration number
NCT00294658
Ethics application status
Date submitted
21/02/2006
Date registered
22/02/2006
Date last updated
23/05/2017
Titles & IDs
Public title
Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy
Query!
Scientific title
A Multi-Center, Single-Blind, Randomized Study Comparing Thymectomy to No Thymectomy in Non-Thymomatous Myasthenia Gravis (MG) Patients Receiving Prednisone
Query!
Secondary ID [1]
0
0
1U01NS042685-01A2
Query!
Secondary ID [2]
0
0
R01NS050733
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Myasthenia Gravis
0
0
Query!
Condition category
Condition code
Inflammatory and Immune System
0
0
0
0
Query!
Autoimmune diseases
Query!
Neurological
0
0
0
0
Query!
Other neurological disorders
Query!
Musculoskeletal
0
0
0
0
Query!
Other muscular and skeletal disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Surgery - thymectomy plus prednisone
Treatment: Drugs - prednisone alone
Active Comparator: Thymectomy plus prednisone - Procedure: Extended Transsternal Thymectomy plus prednisone treatment
Placebo Comparator: Prednisone alone - Drug: prednisone alone protocol
Treatment: Surgery: thymectomy plus prednisone
The thymectomy will be performed as soon as possible after randomization.
Treatment: Drugs: 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.
Query!
Intervention code [1]
0
0
Treatment: Surgery
Query!
Intervention code [2]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Primary outcome [2]
0
0
Time-weighted Average Alternate-day Prednisone Dose (mg) Measured Over 3 Years
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [1]
0
0
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Prednisone Use at Enrollment
Query!
Assessment method [1]
0
0
Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Query!
Timepoint [1]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [2]
0
0
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Sex
Query!
Assessment method [2]
0
0
Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Query!
Timepoint [2]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [3]
0
0
Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Age at Disease Onset
Query!
Assessment method [3]
0
0
Myasthenia Gravis (QMG) test. QMG total scores range from 0 to 39 for a given visit, with higher scores indicating more severe disease.
Query!
Timepoint [3]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [4]
0
0
Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Prednisone Use at Enrollment
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [5]
0
0
Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Sex
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [6]
0
0
Subgroup Analyses of Time-weighted Average Average Alternate-day Prednisone Dose (mg) by Age at Disease Onset
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [7]
0
0
Number of Serious Adverse Events
Query!
Assessment method [7]
0
0
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)
Query!
Timepoint [7]
0
0
baseline to 3 years
Query!
Secondary outcome [8]
0
0
Number of Patients With at Least One Serious Adverse Events
Query!
Assessment method [8]
0
0
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)
Query!
Timepoint [8]
0
0
baseline to 3 years
Query!
Secondary outcome [9]
0
0
Classification of Serious Adverse Events
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
baseline to 3 years
Query!
Secondary outcome [10]
0
0
Hospitalization for Exacerbation of Myasthenia Gravis
Query!
Assessment method [10]
0
0
Query!
Timepoint [10]
0
0
baseline to 2 years and baseline to 3 years
Query!
Secondary outcome [11]
0
0
Cumulative Number of Hospital Days
Query!
Assessment method [11]
0
0
Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)
Query!
Timepoint [11]
0
0
baseline to 3 years
Query!
Secondary outcome [12]
0
0
Reason for Hospitalization According to Medical Dictionary for Regulatory Activities Term
Query!
Assessment method [12]
0
0
Number who had hospitalization: Thymectomy plus prednisone n=15 (out of 66); Prednisone alone n=31 (out of 60)
Query!
Timepoint [12]
0
0
baseline to 3 years
Query!
Secondary outcome [13]
0
0
Time-weighted Average Prescribed Alternate Day Prednisone Dose (mg)
Query!
Assessment method [13]
0
0
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.
Query!
Timepoint [13]
0
0
baseline-day 20, month 1,2, 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [14]
0
0
Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 1: Penalized Using Maximum Dose Before Azathioprine)
Query!
Assessment method [14]
0
0
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.
Query!
Timepoint [14]
0
0
baseline, month 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [15]
0
0
Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 2: Penalized Using Dose at Time of Starting Azathioprine)
Query!
Assessment method [15]
0
0
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.
Query!
Timepoint [15]
0
0
baseline, month 1 , 2 , 3, 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [16]
0
0
Time-Weighted Average MG Activity of Daily Living (MG-ADL)
Query!
Assessment method [16]
0
0
MG Activity of Daily Living total scores range from 0 to 24, with the lower scores indicating better daily living quality of life.
Query!
Timepoint [16]
0
0
baseline, month 4, 6 and every 3 months through 36 months
Query!
Secondary outcome [17]
0
0
Time-Weighted Average MG Activity of Daily Living (MG-ADL) at Month 12, 24, and 36
Query!
Assessment method [17]
0
0
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.
Query!
Timepoint [17]
0
0
Month 12, 24, and 36
Query!
Secondary outcome [18]
0
0
Azathioprine Use
Query!
Assessment method [18]
0
0
Query!
Timepoint [18]
0
0
baseline to 3 years
Query!
Secondary outcome [19]
0
0
Plasma Exchange Use
Query!
Assessment method [19]
0
0
Query!
Timepoint [19]
0
0
baseline to 3 years
Query!
Secondary outcome [20]
0
0
Intravenous Immunoglobulin Use
Query!
Assessment method [20]
0
0
Query!
Timepoint [20]
0
0
baseline to 3 years
Query!
Secondary outcome [21]
0
0
Minimal Manifestation (MM) Status at Month 12, 24 and 36
Query!
Assessment method [21]
0
0
Number of participants who were in minimal manifestation status at month 12, 24 and 36.
Query!
Timepoint [21]
0
0
Month 12, 24 and 36
Query!
Secondary outcome [22]
0
0
Cumulative Days in Hospital for Myasthenia Gravis Exacerbation
Query!
Assessment method [22]
0
0
Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=17 (out of 60)
Query!
Timepoint [22]
0
0
baseline to 2 years
Query!
Secondary outcome [23]
0
0
Cumulative Days in Hospital for Myasthenia Gravis Exacerbation
Query!
Assessment method [23]
0
0
Number of patients with MG exacerbation: Thymectomy plus prednisone=6 (out of 66); Prednisone alone=22 (out of 60)
Query!
Timepoint [23]
0
0
baseline to 3 years
Query!
Secondary outcome [24]
0
0
Short Form-36 Standardized Physical Component
Query!
Assessment method [24]
0
0
Range from 0 to 100, the higher the physical component value, the better the mental health.
Query!
Timepoint [24]
0
0
Month 0, Month 12, Month 24 and Month 36
Query!
Secondary outcome [25]
0
0
Short Form-36 Standardized Mental Component
Query!
Assessment method [25]
0
0
Range from 0 to 100, the higher the mental component value, the better the mental health.
Query!
Timepoint [25]
0
0
Month 0, Month 12, Month 24 and Month 36
Query!
Secondary outcome [26]
0
0
Treatment Associated Complications (TAC)
Query!
Assessment method [26]
0
0
Treatment associated complications measured complications occurred by myasthenia gravis patients. Report number of participant with at least one complications by each visit.
Query!
Timepoint [26]
0
0
Month 0, 1, 2, 3, 4 then every 3 months through Month 36
Query!
Secondary outcome [27]
0
0
Treatment Associated Symptoms (TAS)
Query!
Assessment method [27]
0
0
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.
Query!
Timepoint [27]
0
0
Month 0, 1, 2, 3, 4 then every 3 months through Month 36
Query!
Eligibility
Key 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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
65
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people assessing the outcomes
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/06/2006
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/12/2015
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
126
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
University of Sydney, Royal Prince Alfred Hospital and The University of Sydney - Sydney
Query!
Recruitment hospital [2]
0
0
University of Melbourne, Melbourne, The Royal Melbourne Hospital, Dept of Neurology, Royal Melbourne Hospital - Victoria
Query!
Recruitment postcode(s) [1]
0
0
- Sydney
Query!
Recruitment postcode(s) [2]
0
0
3050 - Victoria
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Alabama
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Arizona
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
California
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Florida
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Georgia
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Indiana
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Kansas
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Massachusetts
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Michigan
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Minnesota
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Missouri
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
New Jersey
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
New York
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
North Carolina
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Ohio
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Texas
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Vermont
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
Virginia
Query!
Country [19]
0
0
United States of America
Query!
State/province [19]
0
0
Washington
Query!
Country [20]
0
0
United States of America
Query!
State/province [20]
0
0
West Virginia
Query!
Country [21]
0
0
United States of America
Query!
State/province [21]
0
0
Wisconsin
Query!
Country [22]
0
0
Argentina
Query!
State/province [22]
0
0
Buenos Aires
Query!
Country [23]
0
0
Brazil
Query!
State/province [23]
0
0
Brasilia
Query!
Country [24]
0
0
Brazil
Query!
State/province [24]
0
0
Curitiba
Query!
Country [25]
0
0
Brazil
Query!
State/province [25]
0
0
Rio De Janeiro
Query!
Country [26]
0
0
Canada
Query!
State/province [26]
0
0
Alberta
Query!
Country [27]
0
0
Canada
Query!
State/province [27]
0
0
British Columbia
Query!
Country [28]
0
0
Canada
Query!
State/province [28]
0
0
Ontario
Query!
Country [29]
0
0
Canada
Query!
State/province [29]
0
0
Quebec
Query!
Country [30]
0
0
Chile
Query!
State/province [30]
0
0
Santiago
Query!
Country [31]
0
0
Germany
Query!
State/province [31]
0
0
Baden-Württemberg
Query!
Country [32]
0
0
Germany
Query!
State/province [32]
0
0
Bavaria
Query!
Country [33]
0
0
Germany
Query!
State/province [33]
0
0
North Rhine-Westphalia
Query!
Country [34]
0
0
Germany
Query!
State/province [34]
0
0
Rhineland-Palatinate
Query!
Country [35]
0
0
Germany
Query!
State/province [35]
0
0
Münster
Query!
Country [36]
0
0
Germany
Query!
State/province [36]
0
0
Tübingen
Query!
Country [37]
0
0
Italy
Query!
State/province [37]
0
0
Milan
Query!
Country [38]
0
0
Italy
Query!
State/province [38]
0
0
Rome
Query!
Country [39]
0
0
Italy
Query!
State/province [39]
0
0
Torino
Query!
Country [40]
0
0
Japan
Query!
State/province [40]
0
0
Ishikawa
Query!
Country [41]
0
0
Japan
Query!
State/province [41]
0
0
Kyushu
Query!
Country [42]
0
0
Mexico
Query!
State/province [42]
0
0
Mexico
Query!
Country [43]
0
0
Netherlands
Query!
State/province [43]
0
0
Leiden
Query!
Country [44]
0
0
Poland
Query!
State/province [44]
0
0
Województwo
Query!
Country [45]
0
0
Portugal
Query!
State/province [45]
0
0
Porto
Query!
Country [46]
0
0
South Africa
Query!
State/province [46]
0
0
Cape Town
Query!
Country [47]
0
0
Spain
Query!
State/province [47]
0
0
Barcelona
Query!
Country [48]
0
0
Taiwan
Query!
State/province [48]
0
0
New Taipei
Query!
Country [49]
0
0
Thailand
Query!
State/province [49]
0
0
Bangkok
Query!
Country [50]
0
0
United Kingdom
Query!
State/province [50]
0
0
Glasgow
Query!
Country [51]
0
0
United Kingdom
Query!
State/province [51]
0
0
Liverpool
Query!
Country [52]
0
0
United Kingdom
Query!
State/province [52]
0
0
Manchester
Query!
Country [53]
0
0
United Kingdom
Query!
State/province [53]
0
0
Oxford
Query!
Country [54]
0
0
United Kingdom
Query!
State/province [54]
0
0
Sheffield
Query!
Funding & Sponsors
Primary sponsor type
Other
Query!
Name
University of Alabama at Birmingham
Query!
Address
Query!
Country
Query!
Other collaborator category [1]
0
0
Government body
Query!
Name [1]
0
0
National Institute of Neurological Disorders and Stroke (NINDS)
Query!
Address [1]
0
0
Query!
Country [1]
0
0
Query!
Ethics approval
Ethics application status
Query!
Summary
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.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT00294658
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Gary Cutter, PhD
Query!
Address
0
0
University of Alabama at Birmingham School of Public Health, Department of Biostatistics
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00294658
Download to PDF