Myasthenia Gravis
Conditions
Interventions
Telitacicept Treatment Group:Telitacicept (recombinant human B lymphocyte stimulator receptor-antibody fusion protein), subcutaneous injection stratified by body weight: 160 mg/time for patients >=40
adjusted to once every 2 weeks from Week 13 based on MSE assessment, in combination with standard treatment
Standard Treatment Control Group (Non-intervention Group):Receive only standard treatment for myasthenia gravis (including cholinesterase inhibitors, glucocorticoids, immunosuppressants, etc.), withou
Sponsors
Tangdu Hospital, The Fourth Military Medical University
Eligibility
Sex/Gender
All
Age
5 Years to 18 Years
Inclusion criteria
Inclusion criteria: Study participants may be enrolled in this study only if all of the following criteria are met: 1. Diagnosis of myasthenia gravis (MG) by a neurologist in accordance with the Chinese Guidelines for the Diagnosis and Treatment of Myasthenia Gravis (2025 Edition); 2. Age of the participant is 5 years to less than 18 years at the time of informed consent/assent; 3. Myasthenia Gravis Foundation of America (MGFA) clinical classification Type I to IVa; 4. Maintenance of any one stable standard treatment regimen as listed below: (1)Cholinesterase inhibitors: stable for at least 1 month prior to enrollment. (2)Glucocorticoids: prednisone dose <= 40 mg/day or equivalent dose of other glucocorticoids (stable for at least 1 month prior to enrollment); (3)Immunosuppressants: mainly including azathioprine, tacrolimus, mycophenolate mofetil (stable for at least 1 month prior to enrollment). (4)Voluntary signing of informed consent by the patient and their parents or legal guardians.
Exclusion criteria
Exclusion criteria: 1. Severe organ dysfunction unrelated to MG: Severe impairment of the central nervous system, respiratory system, circulatory system, digestive system, or urinary system not caused by MG, as determined by an experienced clinician. 2. Any unresolved acute, chronic, or systemic bacterial or other infections that are considered clinically significant by the investigator and for which appropriate antibiotic therapy has not been administered. 3. History of hypersensitivity to human-derived biological products. 4. Use of B-cell depleting agents (e.g., rituximab) within 6 months prior to the first dose. 5. Use of neonatal Fc receptor antagonists or complement inhibitors within 1 month prior to the first dose; use of intravenous immunoglobulin (IVIg) or performance of plasma exchange within 1 month prior to the first dose. 6. Underwent thymectomy within 6 months prior to screening. 7. Patients considered by the investigator to be unsuitable for participation in this trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The proportion of patients achieving minimal symptom expression (MSE) at Week 24; | — |
Secondary
| Measure | Time frame |
|---|---|
| The mean change in ADL scores at week 4, week 8, week 12 and week 24;The mean change in QMG scores at week 4, week 8, week 12 and week 24;The mean change in MG II scores at week 4, week 8, week 12 and week 24;The mean change in EQ-5D-Y scores at week 4, week 8, week 12 and week 24;Proportion of patients with prednisone or equivalent glucocorticoid dose = 2 points on the MG-ADL score or >= 3 points on the QMG score, lasting for more than 24 hours).;Incidence of adverse events (AEs) and serious adverse events (SAEs) during the study period;Changes in height, weight and BMI at baseline (pre-treatment) and Week 24;Changes in key hormone levels (TSH, FT4, IGF-1, as well as LH and FSH in adolescent children) at baseline (pre-treatment) and Week 24; | — |
Countries
China
Contacts
Public ContactChang Ting
Tangdu Hospital, The Fourth Military Medical University
Outcome results
None listed