the safety and efficacy of s103 in the treatment of refractory generalized myasthenia gravis

Conditions: Myasthenia Gravis, Generalized

Summary: this study is a single-center, open-label, single-arm, dose-exploration study to evaluate the safety and preliminary effectiveness of bcma car-t（s103） in the treatment of refractory, generalized myasthenia gravis. the study is a dose escalation trial in adult, refractory, systemic mg patients. a total of 6-24 mg patients who meet the inclusion criteria are expected to be recruited.


Eligibility criteria:
inclusion criteria: 1. age ≥18 years, ≤80 years; 2. mgfa classification type iia-iva; 3. voluntary participation in the study: understanding and awareness of the study and voluntary signing of the informed consent form; 4. meeting the diagnosis of myasthenia gravis; 5. assessed by the investigator as meeting the diagnostic criteria for refractory mg: fulfilling one of the following 4 conditions: 1. after adequate dose and duration of at least 2 conventional immunotherapies (including steroid and non-steroid immunosuppressants), the post-intervention status (pis) is unchanged or worsened. 2. after adequate dose and duration of at least 2 conventional immunotherapies, the pis is improved, but the mg-adl score remains * 6 for at least 6 months. 3. after adequate dose and duration of at least 2 conventional immunotherapies, the pis is remission or improvement, but during regular tapering of immunotherapy, there are still ≥2 exacerbations per year (mg-adl score ≥6). 4. the investigator considers that, despite current conventional immunotherapy, mg still imposes a significant functional burden on the patient. 6. mg-adl score ≥6 or qmg score ≥11 at screening and baseline, with ocular muscle score less than 50% of the total score. exclusion criteria: 1. any medical or psychiatric condition that the investigator deems may endanger the study participant or affect their ability to participate in the study; or any condition the investigator considers associated with poor compliance; 2. females who are breastfeeding or pregnant, or plan to become pregnant at any time during the 12-month period after receiving car-t therapy, or have a history of spontaneous or induced abortion within 4 weeks prior to screening; 3. study participants with clinically relevant active infections (e.g., sepsis, pneumonia, or abscess) or severe infections (requiring hospitalization or antibiotic treatment) within 4 weeks prior to screening; 4. thymectomy within 6 months prior to baseline or planned thymectomy during the study period, or thymoma requiring chemotherapy and/or radiotherapy at any time; 5. study participants who received live attenuated vaccines within 8 weeks prior to screening; or plan to receive live vaccines within 8 weeks after treatment; 6. study participants who received prior rituximab treatment within 6 months before screening; 7. treatment with tocilizumab, eculizumab, or efgartigimod within 3 months prior to screening; 8. intravenous immunoglobulin, plasma exchange, or immunotherapy within 4 weeks prior to screening; 9. known severe underlying diseases, such as liver or kidney dysfunction, hematologic disorders, prior severe cardiovascular diseases, severe hypertension, diabetes, or poorly controlled blood pressure or blood glucose; 10. unresected thymoma; 11. rapid symptom deterioration during the lead-in period, progressing to crisis or pre-crisis state (mgfa ivb-v); 12. other conditions deemed by the investigator as unsuitable for study participation.