Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis (NCT06371040) | Clinical Trial Compass
RecruitingPhase 1
Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis
China9 participantsStarted 2024-07-12
Plain-language summary
This study is a single-center, open-label, single-arm, dose-exploration study to evaluate the safety and preliminary effectiveness of CD19-BCMA CAR-T in the treatment of refractory, generalized myasthenia gravis. The study is a dose escalation trial in adult, refractory, systemic MG patients. The Keyboard method will be used to perform dose escalation to explore the maximum tolerated dose (MTD). A total of 12 MG patients who meet the inclusion criteria are expected to be recruited.
Who can participate
Age range18 Years – 80 Years
SexALL
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Inclusion criteria
✓. Age ≥18 years old and ≤80 years old;
✓. The subject signs the informed consent form, is willing and able to comply with the protocol, complete the research assessment and return for follow-up;
✓. To be diagnosed as a patient with systemic MG, the patient is required to have positive myasthenia-related antibodies (AChR-Ab, Musk-Ab or LRP4) on the basis of typical myasthenic symptoms;
✓. Evaluated by the researcher as refractory MG. Refractory MG is defined as:
✓. Treatment failed after receiving at least 2 immunosuppressants
✓. Definition of treatment failure: 1) Persistent weakness and impairment of daily activities; 2) MG aggravation and/or crisis during treatment; 3) Intolerance to immunotherapy due to side effects or comorbidities;
✓. Repeated plasma exchange (PE) or intravenous immune globulin (IVIg) treatment is required to control symptoms;
✓. The researchers believe that despite the current routine immunotherapy for patients, MG still imposes a large functional burden on patients.
Exclusion criteria
✕. The researcher believes that there is any medical or mental condition that may harm the research participant or affect the research participant's ability to participate in this study; or any condition that the researcher believes is related to poor compliance;
✕. Women who are lactating or pregnant, or women who plan to become pregnant at any time within 12 months after receiving CART treatment, or who have a history of spontaneous abortion or induced abortion within 4 weeks before screening;
What they're measuring
1
Frequency, type, and severity of adverse events
Timeframe: From Baseline (Day 1) to Safety Follow-Up Visit (up to 4 weeks)
✕. Study participants have clinically relevant active infections (such as sepsis, pneumonia or abscess) or serious infections (resulting in hospitalization or requiring antibiotic treatment) within 4 weeks before screening;
✕. thymoma that underwent thymectomy within 6 months before baseline or was planned to undergo thymectomy during the study, or required chemotherapy and/or radiotherapy at any time;
✕. Investigator participants have received live attenuated vaccine vaccination within 8 weeks before screening; or plan to receive live vaccine vaccination within 8 weeks after treatment;
✕. Study participants have received rituximab treatment within 6 months before screening;
✕. Have received tocilizumab or eculizumab treatment within 3 months before screening;
✕. Have received intravenous human immunoglobulin, plasma exchange, or immunotherapy within 4 weeks before screening;