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.
This is an investigator-initiated trial to evaluate the safety and efficacy of universal CD19/BCMA CAR T-cells in Relapsed or Refractory Myasthenia gravis. Study intervention consists of a single infusion of universal CAR T-cells administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide. Interim analysis will be performed when participants finish the visit 90 days after CAR T-cell infusion.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
A single injection of UCAR T-cells, referred to as universal allogeneic anti-CD19/BCMA CAR T-cells
The first affiliated hospital of medical college of zhejiang university
Hangzhou, Zhejiang, China
The number and severity of dose-limiting toxicity (DLT) events
DLT will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, and the ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells.
Time frame: Within 28 Days After UCAR T-cell Infusion
The total number, incidence, and severity of AEs
Time frame: Up to 90 days After UCAR T-cell Infusion
Changes of Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
MG-ADL scale assesses the impact of gMG on daily functions by measuring 8 signs or symptoms that are commonly affected in MG. Each item is measured on a 4-point scale, where a score of 0 represents normal function and a score of 3 represents the loss of ability to perform that function. Total scores range from 0 to 24 points, with a higher score showing more severe MG.
Time frame: Up to 24 Months After UCAR T-cell Infusion
Quantitative Myasthenia Gravis Score (QMG)
The QMG score is a 13-item scale used to quantify disease severity in myasthenia gravis. The scale measures ocular, bulbar, respiratory, and limb function, grading each finding, and ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits).
Time frame: Up to 24 Months After UCAR T-cell Infusion
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