refractory generalized Myasthenia Gravis
Conditions
Interventions
Experimental group:Using SENL103 autologous T-cell injection
Sponsors
Tangdu Hospital, the Fourth Military Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1.Age >=18 years =6 points for at least six months; (3)After receiving adequate doses and full courses of treatment with at least two conventional immunotherapy drugs, and having undergone treatment with at least one biological targeted drug, the Post-Intervention Status (PIS) shows remission or improvement, but there are still >=2 exacerbations of disease symptoms (MG-ADL score >=6) per year; 6.MG-ADL score >=6 or QMG score >=11 at screening and baseline, with ocular muscle score less than 50% of the total score; 7.Male study participants must agree to use contraception during the treatment period and for 2 years after the last dose of the study treatment, and must also agree not to donate sperm throughout the entire study period; 8.If the participant is a woman of childbearing potential (WOCBP), she must agree to use contraception during the treatment period and for 2 years after the last dose of the study treatment. At screening, the participant’s serum pregnancy test result must be negative; before the first dose of the study drug at baseline, the urine pregnancy test result must be confirmed negative.
Exclusion criteria
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 2 years 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 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.Participants with a positive immunological test for Mycobacterium tuberculosis can be enrolled if they meet the following conditions simultaneously:(1)No radiological evidence of active tuberculosis.(2)No signs of tuberculosis intoxication (such as low-grade fever, fatigue, night sweats, decreased appetite, weight loss, etc.), or respiratory symptoms (such as cough, sputum production for more than 2 weeks, or with hemoptysis, blood-streaked sputum); 12.Participants who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and have a quantitative level of hepatitis B virus (HBV) DNA in peripheral blood above the lower limit of detection; participants who are positive for hepatitis C virus (HCV) antibody and have a quantitative level of HCV RNA above the lower limit of detection; participants who are positive for human immunodeficiency virus (HIV) antibody; participants who are positive for cytomegalovirus (CMV) DNA; participants who are positive for both specific and non-specific antibodies to Treponema pallidum (the causative agent of syphilis); 13.Other circumstances in which the investigator considers the participant to be ineligible for enrollment in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Types and incidence of dose-limiting toxicities (DLTs) within 28 days after reinfusion;Types, frequency and severity of adverse events (AEs) within 28 days after reinfusion; | — |
Secondary
| Measure | Time frame |
|---|---|
| Post-reinfusion 28-day, 3-month, and 6-month MG-ADL, QMG, MGC, and QOL15 baseline-to-post-treatment change;Proportion of participants achieving MSE status (MG-ADL =2-point reduction in MG-ADL/QMG scores from baseline to post-treatment, and symptom fluctuation patterns at these time points; | — |
Countries
China
Contacts
Public ContactChang Ting
Tangdu Hospital, the Fourth Military Medical University
Outcome results
None listed