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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years - 80 Years
Gender All
More Inclusion & Exclusion 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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT06958939
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Ting Chang, MD
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Ting Chang
Principal Investigator Affiliation The Second Affiliated Hospital of Air Force Medical University
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Not yet recruiting
Countries China
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Myasthenia Gravis, Generalized
Arms & Interventions

Arms

Experimental: S103 CAR-T

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

Interventions

Drug: - Dose level 1 group

1.0 × 10e6/kg S103 CAR-T cells

Drug: - Dose level 2 group

2.0 × 10e6/kg S103 CAR-T cells

Drug: - Dose level 3 group

4.0 × 10e6/kg S103 CAR-T cells

Drug: - Dose level 4 group

8.0 × 10e6/kg S103 CAR-T cells

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

International Sites

Xi'an 1790630, Shaanxi 1796480, China

Status

Address

The Second Affiliated Hospital of Air Force Medical University

Xi'an 1790630, Shaanxi 1796480, 710038

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