Status:
NOT_YET_RECRUITING
The Safety and Efficacy of S103 in the Treatment of Refractory Generalized Myasthenia Gravis
Lead Sponsor:
Ting Chang, MD
Collaborating Sponsors:
Hebei Senlang Biotechnology Co., LTD
Conditions:
Myasthenia Gravis, Generalized
Eligibility:
All Genders
18-80 years
Phase:
PHASE1
Brief 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 myast...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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;
- 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;
- Unresected thymoma;
- Rapid symptom deterioration during the lead-in period, progressing to crisis or pre-crisis state (MGFA IVb-V);
- Other conditions deemed by the investigator as unsuitable for study participation.
Key Trial Info
Start Date :
May 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 30 2026
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT06958939
Start Date
May 1 2025
End Date
August 30 2026
Last Update
May 6 2025
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
The Second Affiliated Hospital of Air Force Medical University
Xi'an, Shaanxi, China, 710038