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This is a 6 month study that will evaluate the efficacy of 5mg tofacitinib tablet taken twice a day in patients with refractory myasthenia gravis. Twenty adult participants will receive a four month treatment of tofacitinib followed by elution for one month.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| tofacitinib 5mg twice a day | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tofacitinib 5 MG | Drug | tofacitinib 5 mg tablet administered twice a day for 6 months |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6 | A quantitative MG scoring system (QMG Score) is essential in the objective evaluation of therapy for MG.This scoring system is based on quantitative testing of sentinel muscle groups. Total QMG score range from 0 (extreme disease severity) to 39 (none), higher score indicated less disease severity. | Baseline, Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6 | The myasthenia gravis activities of daily living (MG-ADL) profile is an eight-item patient-reported scale developed to assess MG symptoms and their effects on daily activities.Total MG-ADL score range from 0 (none) to 24 (extreme disease conditions), higher score indicated more disease severity. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Serum Titers of anti-acetylcholine receptor antibodies at Week 16 | Anti-acetylcholine receptor antibodies are the major pathological autoantibodies of MG. For anti-AChR antibodies-positive candidates, we followed the antibodies titers at Week 16. | Baseline, Week 16 |
| Change From Baseline in Inflammatory Cytokines at Week 12 |
Inclusion Criteria:
meet all of the criteria in above as well as at least one of the below:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Huashan Hospital | Shanghai | 200040 | China |
Individual participant data (IPD) are not available to other researchers.
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| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| C479163 | tofacitinib |
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| Baseline, Month 6 |
| Change From Baseline in Myasthenia Gravis Composite (MGC) Scores at Month 6 | The MGC score consists of test items that measure symptoms and signs of MG, with weighted response options. Total MGC score range from 0 (none) to 50 (extreme muscle weakness), higher score indicated more disease severity. | Baseline, Month 6 |
| Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6 | The MG-QOL15 is helpful in informing the clinician about the patient's perception of the extent of and dissatisfaction with MG-related dysfunction. Total MG-QOL15 score range from 0 (none) to 60 (extreme disease severity), higher score indicated more disease severity. | Baseline, Month 6 |
| Change From Baseline in Doses of Steroids at Month 6 | Steroids is recommended as the first-line treatment for myasthenia gravis. Here we followed the combined doseage of steroids while administrating tofacitinib for enrolled participants. | Baseline, Month 6 |
MG is an autoimmune disease manifested by impairment at the neuromuscular junction featured by weakness and fatigability. We compared the inflammatory cytokines (IL-6, IL-10, IL-17A, and IL-23) at Baseline and Week 12 |
| Baseline, Week 12 |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |