Myasthenia gravis
Conditions
Interventions
The Tacrolimus group: In the Tacrolimus group, subjects received oral treatment with a daily dose of 3mg of Tacrolimus.
Sponsors
Peking University People's Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: Criteria for Inclusion: (1) Voluntary signing of informed consent by the patient; (2) Age between 18 and 80 years, regardless of gender; (3) Diagnosis of generalized myasthenia gravis (MG), with onset of symptoms within less than 6 months, meeting the following conditions: Weakness in specific striated muscle groups with patchy distribution, demonstrating fluctuation and fatigability; symptoms of weakness are typically worse in the morning and improve after rest; Positive results in the neostigmine test (Xu's quantitative evaluation method); and/or electromyography (EMG) findings: repetitive nerve stimulation (RNS) showing a decrement of at least 10% in amplitude with low-frequency stimulation in at least 1 nerve, or single-fiber EMG (SFEMG) showing two or more "jitter" abnormalities; Exclusion of other syndromes causing weakness such as Guillain-Barré syndrome, Lambert-Eaton myasthenic syndrome, etc.; Time since diagnosis of generalized MG = 6 months; (4) Serum testing positive for AChR antibodies; (5) Classification according to the Myasthenia Gravis Foundation of America (MGFA) clinical types II (including IIa and IIb), III (including IIIa and IIIb), or IVa; (6) Myasthenia Gravis Activities of Daily Living (MG-ADL) scale score = 6, with ocular-related score less than 50% of the total score; (7) Quantitative Myasthenia Gravis (QMG) score = 8, with at least 2 points in = 4 items; (8) Maintenance of stable standard treatment regimen, defined as stable use of any of the following (alone or in combination): a. Acetylcholinesterase inhibitors: maintained at a stable dose = 480mg/day for at least 2 weeks before the start of the clinical trial; b. Glucocorticoids: prednisone dose = 15mg and = 60mg/day or equivalent dose of other steroids, maintained at a stable dose for at least 1 month before the start of the clinical trial.
Exclusion criteria
Exclusion criteria: Exclusion Criteria: (1) Presence of other autoimmune diseases such as systemic lupus erythematosus, rheumatoid arthritis, Sjögren's syndrome, etc., although patients with Graves' disease and hypothyroidism, as evaluated by the investigator, may be exempted; (2) Significant laboratory abnormalities (e.g., hemoglobin 9.0% or fasting blood glucose = 11.1 mmol/L; (13) Patients currently diagnosed with thymoma-associated immunodeficiency syndrome (Good syndrome) or those who have undergone thymectomy within 6 months prior to screening; (14) Receipt of any live vaccine or COVID-19 vaccine (including nasal spray) within 3 months prior to the start of the trial or planned during the study period; (15) Patients with malignant tumors; (16) Allergy to human-derived biologics; (17) Participation in any other clinical trial within 28 days prior to the start of the clinical trial or within 5 half-lives of investigational drugs from previous clinical trial participation (whichever is longer); (18) Pregnant or lactating women and patients planning conception during the trial period; (19) Known alcohol or substance abuse/addiction that may impact compliance with trial requirements; (20) Patients deemed unsuitable for participation in the trial by the investigator (e.g., patients with severe mental disorders).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The proportion of patients with QMG scores = 4 at 24 weeks daily prednisone dose = 10mg, and no rescue therapy; | — |
Secondary
| Measure | Time frame |
|---|---|
| The change in MG-ADL score from baseline at Weeks 12 and 24 ;The change in MGFA-QMG score from baseline at Weeks 12 and 24 ;The change in MG-QOL15r score from baseline at Weeks 12 and 24 ;The proportion of subjects with a decrease of =3 points in MG-ADL score from baseline at Weeks 12 and 24 ;The proportion of subjects with a decrease of =5 points in MGFA-QMG score from baseline at Weeks 12 and 24 ;The percentage of subjects experiencing treatment-related adverse reactions; | — |
Countries
China
Contacts
Public ContactZhaoxu Zhang
Peking University People's Hospital
Outcome results
None listed