UCAR T-cell Therapy Targeting CD19/BCMA in Patients With r/r Myasthenia Gravis

Launched by ZHEJIANG UNIVERSITY · Apr 16, 2025

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ClinConnect Summary

This clinical trial, called UCAR T-cell Therapy, is exploring a new treatment for patients with relapsed or refractory Myasthenia Gravis, a condition that affects muscle strength and can make everyday activities difficult. The trial will test a special type of immune cell therapy designed to target specific proteins (CD19 and BCMA) involved in the disease. It will involve up to 18 participants aged 18 and older who have already tried standard treatments but continue to struggle with their symptoms.

To be eligible, participants must have tested positive for certain markers in their blood and have a specific level of muscle weakness as measured by a standard scoring system. They should also have been receiving treatment with immunosuppressant medications for over a year. If you or someone you know is interested in this trial, it's important to know that the study is not yet recruiting participants. Those who join can expect close monitoring and support throughout the treatment process, but there are strict guidelines to ensure the safety and health of everyone involved.

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Eligibility criteria

• Inclusion Criteria:
• • 1. Age ≥ 18 years
• • 2. Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
• • 3. Patients with relapsed refractory myasthenia gravis (MG) who have positive abnormal antibodies, the total score of MG-ADL ≥5, and the eye muscle score \< 50% of the total score; It is classified as Grade II-IV according to the 2020 MGFA diagnostic criteria.
• • 4. Specific requirements include: i. Receiving standardized treatment with at least one immunosuppressant for more than 1 year, and having any of the following malcontrolled conditions: 1) persistent inability to affect daily life; 2) Aggravation of MG symptoms and/or crisis episodes occur despite standard treatment; 3) Inability to tolerate immunosuppressive therapy. ii. plasma exchange or maintenance of intravenous immunoglobulin therapy is required.
• 5. Functional requirements for major organs are as follows:
• • 1. The bone marrow function needs to meet: a Neutrophil count ≥ 1.5× 10 \^ 9/L; b. Hemoglobin ≥90g/L: c. Platelets ≥ 80 × 10 \^ 9/L.
• • 2. Liver function: ALT ≤ 3 × UL; AST ≤ 3×ULN# Total bilirubin ≤ 2.0 ×ULN (excluding Gilbert syndrome, total bilirubin ≤ 3.0 × ULN).
• • 3. Renal function: creatinine clearance rate (CrCl) ≥ 30 ml/min(Cockcroft/Gault formula, excluding acute CrCl decline caused by the disease itself).
• • 6. Female subjects of childbearing potential and male subjects with partners of childbearing potential must use medically approved contraception or abstinence during the study treatment period and for at least 6 months after the end of the study treatment; Female subjects of childbearing potential must have a negative Human chorionic gonadotropin (HCG) test within 7 days before study enrollment and not be lactating.
• • 7. Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.
• Exclusion Criteria:
• • 1. Subjects with a history of severe drug allergies or allergic tendencies.
• • 2. Presence or suspicion of uncontrolled or treatment-required fungal,bacterial, viral, or other infections.
• • 3. Subjects with central nervous system diseases caused by autoimmune diseases or non-autoimmune diseases (including epilepsy, psychosis, organic brain syndrome, cerebral vascular accidents, encephalitis, central nervous system vasculitis).
• • 4. Subjects with insufficient cardiac function.
• • 5. Subjects with congenital immunoglobulin deficiencies.
• • 6. History of malignancy within five years.
• • 7. Subjects with end-stage renal failure.
• • 8. Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA titer higher than the upper limit of detection; subjects positive for hepatitis C virus (HCV) antibody and peripheral blood HCV RNA; individuals positive for human immunodeficiency virus (HIV) antibody; individuals positive for syphilis testing.
• • 9. Subjects with psychiatric disorders and severe cognitive impairments.
• • 10. Subjects who had participated in other clinical trials within 3 months prior to enrollment.
• • 11. Subjects who have used immunosuppressive agents or biologics with therapeutic effects on the disease within five half-life before enrollment
• • 12. Pregnant women or women planning to conceive
• • 13. Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study.