The Safety and Efficacy of S103 in the Treatment of Refractory Generalized Myasthenia Gravis

Korábbi kezelés

• 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;

Más betegség

• 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;
• Other conditions deemed by the investigator as unsuitable for study participation.

Laborértékek

• 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;

Egyéb

• Unresected thymoma;
• Rapid symptom deterioration during the lead-in period, progressing to crisis or pre-crisis state (MGFA IVb-V);

• 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
  • 6 for at least 6 months.
  • 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).
  • The investigator considers that, despite current conventional immunotherapy, MG still imposes a significant functional burden on the patient.
• MG-ADL score ≥6 or QMG score ≥11 at screening and baseline, with ocular muscle score less than 50% of the total score.