The Safety and Efficacy of S103 in the Treatment of Refractory Generalized Myasthenia Gravis (NCT06958939) | Clinical Trial Compass
Not Yet RecruitingPhase 1
The Safety and Efficacy of S103 in the Treatment of Refractory Generalized Myasthenia Gravis
China24 participantsStarted 2025-05-01
Plain-language 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.
Who can participate
Age range18 Years – 80 Years
SexALL
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Inclusion criteria
✓. Age ≥18 years, ≤80 years;
✓. MGFA classification type IIa-IVa;
✓. Voluntary participation in the study: understanding and awareness of the study and voluntary signing of the informed consent form;
✓. Meeting the diagnosis of myasthenia gravis;
✓. Assessed by the investigator as meeting the diagnostic criteria for refractory MG: fulfilling one of the following 4 conditions:
✓. 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.
✓. After adequate dose and duration of at least 2 conventional immunotherapies, the PIS is improved, but the MG-ADL score remains
✓. 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).
Exclusion criteria
✕. 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;
✕. 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;
✕. 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;
✕. Thymectomy within 6 months prior to baseline or planned thymectomy during the study period, or thymoma requiring chemotherapy and/or radiotherapy at any time;
✕. Study participants who received live attenuated vaccines within 8 weeks prior to screening; or plan to receive live vaccines within 8 weeks after treatment;
✕. Study participants who received prior rituximab treatment within 6 months before screening;
✕. Treatment with tocilizumab, eculizumab, or efgartigimod within 3 months prior to screening;
✕. Intravenous immunoglobulin, plasma exchange, or immunotherapy within 4 weeks prior to screening;