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

NCT ID: NCT06958939

Last Updated: 2025-05-06

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-01
• Study Completion Date: 2026-08-30

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 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.

Conditions

Myasthenia Gravis, Generalized

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

S103 CAR-T

This study adopted a single-arm design, with all patients receiving sequential administration of different doses of S103 CAR-T

Group Type: EXPERIMENTAL

Dose level 1 group

Intervention Type: DRUG

1.0 × 10e6/kg S103 CAR-T cells

Dose level 2 group

Intervention Type: DRUG

2.0 × 10e6/kg S103 CAR-T cells

Dose level 3 group

Intervention Type: DRUG

4.0 × 10e6/kg S103 CAR-T cells

Dose level 4 group

Intervention Type: DRUG

8.0 × 10e6/kg S103 CAR-T cells

Interventions

Dose level 1 group

1.0 × 10e6/kg S103 CAR-T cells

Intervention Type: DRUG

Dose level 2 group

2.0 × 10e6/kg S103 CAR-T cells

Intervention Type: DRUG

Dose level 3 group

4.0 × 10e6/kg S103 CAR-T cells

Intervention Type: DRUG

Dose level 4 group

8.0 × 10e6/kg S103 CAR-T cells

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hebei Senlang Biotechnology Co., LTD

UNKNOWN

Sponsor Role: collaborator

Ting Chang, MD

OTHER

Sponsor Role: lead

Responsible Party

Ting Chang, MD

Associate Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Ting Chang

Role: STUDY_CHAIR

The Second Affiliated Hospital of Air Force Medical University

Locations

The Second Affiliated Hospital of Air Force Medical University

Xi'an, Shaanxi, China

Countries

China

Central Contacts

Zhe Ruan

Role: CONTACT

ruanzhe1988@fmmu.edu.cn

86 29 84777741

Facility Contacts

Role: primary

changting1981@163.com

02984778845

Other Identifiers

CART-20250321

Identifier Type: -

Identifier Source: org_study_id