NCT06371040 Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis
| NCT ID | NCT06371040 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Ting Chang, MD |
| Condition | Myasthenia Gravis |
| Study Type | INTERVENTIONAL |
| Enrollment | 9 participants |
| Start Date | 2024-07-12 |
| Primary Completion | 2025-11-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 9 participants in total. It began in 2024-07-12 with a primary completion date of 2025-11-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * Study participants will be selected for this study only if they meet all of the following criteria: 1. Age ≥18 years old and ≤80 years old; 2. 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; 3. 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; 4. Evaluated by the researcher as refractory MG. Refractory MG is defined as: 1. Treatment failed after receiving at least 2 immunosuppressants 2. 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; 3. Repeated plasma exchange (PE) or intravenous immune globulin (IVIg) treatment is required to control symptoms; 4. The researchers believe that despite the current routine immunotherapy for patients, MG still imposes a large functional burden on patients. 5. MGFA classification IIa\~IVa at screening and baseline; 6. QMGS score ≥11 points or MG-ADL score ≥5 points at screening and baseline, of which the eye score accounts for no more than 50%; 7. Male study participants must agree to take contraceptive measures during the treatment period and within 1 year after receiving study treatment, and are prohibited from donating sperm throughout the study period; 8. If you are a woman of childbearing potential (WOCBP), you must agree to take contraceptive measures during treatment and for at least 1 year after receiving study treatment. Participants must have a negative serum pregnancy test result during screening; a negative urine pregnancy test result must be confirmed before receiving CART for the first time. Exclusion Criteria: * Prior to screening and the baseline visit, study participants will not be eligible for inclusion in the study if they meet any of the following criteria: 1. 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; 2. 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; 3. 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; 4. 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; 5. 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; 6. Study participants have received rituximab treatment within 6 months before screening; 7. Have received tocilizumab or eculizumab treatment within 3 months before screening; 8. Have received intravenous human immunoglobulin, plasma exchange, or immunotherapy within 4 weeks before screening; 9. Those with known serious underlying diseases, such as liver and kidney damage, blood diseases, previous severe cardiovascular disease, severe hypertension, diabetes, and poor blood pressure and blood sugar control; 10. Unresected thymoma (Note: Subjects with benign thymoma resected more than one year before screening are eligible. Benign is defined as no known metastasis on pathological examination and no intracystic or extracystic Extension. Imaging studies must be performed during the screening period to assess thymic status). 11. Any of the following laboratory abnormalities occur during the screening period (one repeat measurement can be performed during the screening period before randomization to confirm the results) 1. Elevated liver enzymes (aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 3 times the upper limit of normal (ULN)). 2. Total bilirubin\>1.5 times ULN 3. Estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73 m2 4. Abnormal PT or INR, or prolonged APTT \>1.5 times ULN 5. Neutrophil count \<1000cell/ul 6. Platelet count \<50000/mm3 7. Hemoglobin\<8.0g/dl 12. Those with a high-risk history of tuberculosis infection or acquired tuberculosis infection; 13. Known immunodeficiency diseases, including human immunodeficiency virus (HIV) infection; 14. Positive for hepatitis B surface antigen (HBsAg) during the screening period; 15. Receive blood transfusion treatment 4 weeks before screening or during the screening period; 16. Symptoms worsen rapidly during the lead-in period and enter crisis or pre-crisis state (MGFA IVb-V) 17. Other circumstances in which the researcher deems it inappropriate to participate in the study.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06371040 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 80 Years, studying Myasthenia Gravis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06371040 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06371040 currently recruiting?
Yes, NCT06371040 is actively recruiting participants. Contact the research team at ruanzhe573291596@126.com for enrollment information.
Where is the NCT06371040 trial being conducted?
This trial is being conducted at Xi'an, China.
Who is sponsoring the NCT06371040 clinical trial?
NCT06371040 is sponsored by Ting Chang, MD. The trial plans to enroll 9 participants.