Actively Recruiting
Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis
Led by Ting Chang, MD · Updated on 2025-09-15
9
Participants Needed
1
Research Sites
124 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to 80 years old
- Signed informed consent and willingness to comply with the study protocol and follow-up
- Diagnosed with systemic myasthenia gravis with positive antibodies (AChR-Ab, Musk-Ab, or LRP4) and typical symptoms
- Diagnosed with refractory myasthenia gravis defined by failure of at least 2 immunosuppressants, need for plasma exchange or IVIg, or intolerance to immunotherapy
- MGFA classification IIa to IVa at screening and baseline
- QMGS score 11 or higher or MG-ADL score 5 or higher at screening and baseline, with eye symptoms accounting for no more than 50%
- Male participants must agree to use contraception during treatment and for 1 year after, and must not donate sperm during the study
- Women of childbearing potential must agree to contraception during treatment and for at least 1 year after, with negative pregnancy tests before and during screening
You will not qualify if you...
- Any medical or mental condition that may harm the participant or affect study participation or compliance
- Women who are pregnant, lactating, planning pregnancy within 12 months after treatment, or with recent abortion history within 4 weeks before screening
- Active or serious infections within 4 weeks before screening
- Thymoma with recent or planned thymectomy or requiring chemotherapy or radiotherapy
- Receipt of live attenuated vaccines within 8 weeks before screening or planned within 8 weeks after treatment
- Rituximab treatment within 6 months before screening
- Tocilizumab or eculizumab treatment within 3 months before screening
- Intravenous immunoglobulin, plasma exchange, or immunotherapy within 4 weeks before screening
- Serious underlying diseases including liver, kidney, blood, cardiovascular diseases, severe hypertension, diabetes with poor control
- Unresected thymoma or recent benign thymoma with metastasis or extension
- Laboratory abnormalities including elevated liver enzymes, bilirubin, impaired kidney function, abnormal clotting tests, low neutrophils, low platelets, or low hemoglobin
- High-risk or active tuberculosis infection
- Known immunodeficiency including HIV infection
- Positive hepatitis B surface antigen during screening
- Blood transfusion within 4 weeks before or during screening
- Rapid worsening symptoms leading to crisis or pre-crisis state (MGFA IVb-V)
- Any other condition deemed inappropriate by the researcher
AI-Screening
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Trial Site Locations
Total: 1 location
1
Tangdu Hospital, The Fourth Military Medical University
Xi'an, Shaanxi, China, 710038
Actively Recruiting
Research Team
Z
Zhe Ruan
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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