Phase 3
N=126
Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy
Myasthenia Gravis
Bottom Line
View on ClinicalTrials.gov: NCT00294658 ↗Enrolled (actual)
126
Serious AEs
46.0%
Results posted
Jan 2017
Primary outcome: Primary: Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years — 6.15; 8.99 units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- thymectomy plus prednisone (Procedure); prednisone alone (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- University of Alabama at Birmingham
- Primary completion
- Nov 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years |
6.15; 8.99 | — |
| PRIMARY Time-weighted Average Alternate-day Prednisone Dose (mg) Measured Over 3 Years |
32; 54 | — |
| SECONDARY Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Prednisone Use at Enrollment |
6.30; 9.10; 5.66; 8.84 | — |
| SECONDARY Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Sex |
6.47; 9.73; 5.23; 7.45 | — |
| SECONDARY Subgroup Analyses of Time-weighted Average Quantitative Myasthenia Gravis Score by Age at Disease Onset |
6.50; 9.60; 5.33; 7.85 | — |
| SECONDARY Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Prednisone Use at Enrollment |
35; 56; 25; 45 | — |
| SECONDARY Subgroup Analyses of Time-weighted Average Alternate-day Prednisone Dose (mg) by Sex |
33; 54; 31; 55 | — |
| SECONDARY Subgroup Analyses of Time-weighted Average Average Alternate-day Prednisone Dose (mg) by Age at Disease Onset |
35; 55; 27; 49 | — |
| SECONDARY Number of Serious Adverse Events |
48; 93 | — |
| SECONDARY Number of Patients With at Least One Serious Adverse Events |
25; 33 | — |
| SECONDARY Classification of Serious Adverse Events |
1; 7; 8; 2; 9; 5 | — |
| SECONDARY Hospitalization for Exacerbation of Myasthenia Gravis |
6; 17; 6; 22 | — |
| SECONDARY Cumulative Number of Hospital Days |
8.4; 19.2 | — |
| SECONDARY Reason for Hospitalization According to Medical Dictionary for Regulatory Activities Term |
2; 2; 0; 1; 4; 7 | — |
| SECONDARY Time-weighted Average Prescribed Alternate Day Prednisone Dose (mg) |
34.3; 55.6 | — |
| SECONDARY Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 1: Penalized Using Maximum Dose Before Azathioprine) |
34.4; 64.4 | — |
| SECONDARY Penalized Time-weighted Average Alternative Day Prednisone Dose (mg; Method 2: Penalized Using Dose at Time of Starting Azathioprine) |
33.3; 57.9 | — |
| SECONDARY Time-Weighted Average MG Activity of Daily Living (MG-ADL) |
2.24; 3.41 | — |
| SECONDARY Time-Weighted Average MG Activity of Daily Living (MG-ADL) at Month 12, 24, and 36 |
1.92; 3.33; 2.02; 3.11; 2.14; 2.69 | — |
| SECONDARY Azathioprine Use |
11; 28 | — |
| SECONDARY Plasma Exchange Use |
10; 9 | — |
| SECONDARY Intravenous Immunoglobulin Use |
11; 23 | — |
| SECONDARY Minimal Manifestation (MM) Status at Month 12, 24 and 36 |
41; 20; 39; 20; 39; 24 | — |
| SECONDARY Cumulative Days in Hospital for Myasthenia Gravis Exacerbation |
8.7; 22.5 | — |
| SECONDARY Cumulative Days in Hospital for Myasthenia Gravis Exacerbation |
8.7; 22.5 | — |
| SECONDARY Short Form-36 Standardized Physical Component |
41.4; 37.9; 48.4; 44.4; 50.3; 43.0 | — |
| SECONDARY Short Form-36 Standardized Mental Component |
49.1; 46.2; 39.1; 41.7; 49.9; 46.7 | — |
| SECONDARY Treatment Associated Complications (TAC) |
22; 17; 15; 15; 22; 20 | — |
| SECONDARY Treatment Associated Symptoms (TAS) |
63; 53; 55; 52; 61; 53 | — |
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.
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.
Data sourced from ClinicalTrials.gov (NCT00294658). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.