UCAR T-cell Therapy Targeting CD19/BCMA in Patients With r/r Myasthenia Gravis (NCT06933563) | Clinical Trial Compass
Not Yet RecruitingEarly Phase 1
UCAR T-cell Therapy Targeting CD19/BCMA in Patients With r/r Myasthenia Gravis
China18 participantsStarted 2025-04
Plain-language summary
This is an open label, single-site, dose-escalation study in up to 18 participants with relapsed or refractory Myasthenia gravis. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Age ≥ 18 years
✓. Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
✓. 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.
✓. 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.
✓. Functional requirements for major organs are as follows:
✓. 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.
✓. Liver function: ALT ≤ 3 × UL; AST ≤ 3×ULN# Total bilirubin ≤ 2.0 ×ULN (excluding Gilbert syndrome, total bilirubin ≤ 3.0 × ULN).
✓. Renal function: creatinine clearance rate (CrCl) ≥ 30 ml/min(Cockcroft/Gault formula, excluding acute CrCl decline caused by the disease itself).
Exclusion criteria
✕. Subjects with a history of severe drug allergies or allergic tendencies.
✕
What they're measuring
1
The number and severity of dose-limiting toxicity (DLT) events
Timeframe: Within 28 Days After UCAR T-cell Infusion
2
The total number, incidence, and severity of AEs
Timeframe: Up to 90 days After UCAR T-cell Infusion
3
Changes of Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
Timeframe: Up to 24 Months After UCAR T-cell Infusion
4
Quantitative Myasthenia Gravis Score (QMG)
Timeframe: Up to 24 Months After UCAR T-cell Infusion
. Presence or suspicion of uncontrolled or treatment-required fungal,bacterial, viral, or other infections.
✕. 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).
✕. Subjects with insufficient cardiac function.
✕. Subjects with congenital immunoglobulin deficiencies.
✕. History of malignancy within five years.
✕. Subjects with end-stage renal failure.
✕. 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.