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The goal of this clinical trial is to evaluate the efficacy and safety of Blinatumomab in the treatment of refractory myasthenia gravis, with the expectation of offering a new therapeutic option for refractory patients. The main questions it aims to answer are:
Participants will:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Blinatumomab | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Blinatumomab | Drug | Blinatumomab is used at its starting dose of 9 µg per day and administered as continuous infusion for 5 days (total dose of 38.5 µg). After a 1-week pause, the patients receive a second 5-day infusion with blinatumomab of total 38.5 µg of the drug. |
| Measure | Description | Time Frame |
|---|---|---|
| Change of Myasthenia Gravis Activities of Daily Living (MG-ADL) Score from baseline | The MG-ADL scale is an 8-item questionnaire. It is completed by trained interviewers based on the patients' subjective responses. The total score ranges from 0 to 24, with higher scores indicating greater impact of the disease on daily living activities. | From baseline to 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change of Quantitative Myasthenia Gravis (QMG) Scores from baseline | The QMG scale is a 13-item scale used to objectively assess muscle strength and fatigue in patients with myasthenia gravis. The total score ranges from 0 to 39, with higher scores indicating greater disease severity. | From baseline to 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yuwei Da, M.D. | Contact | 00-86-010-83198493 | dayuwei100@hotmail.com |
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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 |
|---|---|
| C510808 | blinatumomab |
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| Time to achievement of minimal symptom expression (MSE) |
The MSE is a quantifiable, patient-reported outcome measure that assesses treatment goals in myasthenia gravis. It is defined as achieving an MG-ADL score of ≤1, indicating that clinical symptoms of MG are at their minimal level. |
| From baseline to 6 months |
| Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from baseline | The MG-QOL15 scale is a 15-item questionnaire designed to assess the impact of myasthenia gravis on patients' daily life, physical function, social well-being, and mental health. The total score ranges from 0 to 60, with higher scores indicating poorer quality of life. | From baseline to 6 months |
| Change of Myasthenia Gravis Composite (MGC) scores from baseline | The MGC scale is a 10-item scale that combines patient self-assessment and physician examination results. Each item is assigned a weight based on factors such as health risks, quality of life, and prognosis. The total score of the scale is 50 points, with higher scores indicating more severe disease. | From baseline to 6 months |
| Change of antibody titers from baseline | MG antibodies are detected at enrollment and the titers of antibodies will be monitored monthly. | From baseline to 6 months |
| Treatment-Related Adverse Events (AEs) | Record all AEs reported by patients during the study period. | From baseline to 6 months |
| 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 |