This is a multicenter, open-label, dose-escalation/expansion phase 1 study to evaluate the safety, tolerability, pharmacokinetic/pharmacodynamic characteristics and determine the recommended dose of KQ-2003 CAR T-cells for patients with Relapsed/Refractory POEMS Syndrome
Age ≥18 years old, male or female;
Diagnosis of POEMS syndrome with relapsed or refractory disease;
Eastern Cooperative Oncology Group (ECOG) Performance ≤2 ;
Adequate venous access for the apheresis of peripheral blood mononuclear cell;
Subjects who had previously received BCMA-CD19 dual-target CAR-T cell products or autologous stem cell transplantation within 12 weeks before the collection of peripheral blood mononuclear cells;
Known allergy or hypersensitivity reactions to cyclophosphamide, fludarabine, dimethyl sulfoxide (DMSO), CD19, or BCMA-targeted drugs;
Received any treatment that might influence the activity of CAR-T cells prior to the collection of peripheral blood mononuclear cells;
Have history of vaccination within the 4 weeks preceding the collection of peripheral blood mononuclear cells;
Sequential
KQ-2003 CAR T-cells
Biological/VaccineKQ-2003 CAR T-cell therapy involves autologous chimeric antigen receptor T-cells, capable of targeting both human B cell maturation antigen (anti-BCMA CAR) and CD19 antigen molecules (anti-CD19 CAR) simultaneously as a cellular therapy.
Number of patients with dose-limiting toxicity (DLT)
For DLT evaluation, severity (grade) is classified according to common terminology criteria for adverse events version 5.0 (CTCAE v5.0).
Adverse Event
Safety will be assessed by adverse events (AEs), which include clinically significant abnormalities identified during a medical test (e.g. laboratory tests, electrocardiogram, vital signs, physical examinations). AEs will be coded by Medical Dictionary for Regulatory Activities (MedDRA) and their severity will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0).
Maximum Tolerated Dose (MTD)
At least 6 subjects in the MTD dose group must complete the DLT assessment.
Recommended Phase 2 Dose (RP2D)
To determine after all subjects in the Phase 1 dose-escalation study completed DLT observation
Response of serum vascular endothelial growth factor level(VEGF)
The improvements of serum VEGF will be assessed every three months by evaluating changes from baseline and will be described descriptively.
Hematologic response
Hematologic response is also a criterion for assessing the efficacy of treatment for POEMS syndrome. The improvements of serum protein electrophoresis (SPEP) and immunofixation electrophoresis (IFE) will be assessed every three months by evaluating changes from baseline and will be described descriptively.
Response of positron emission tomography-scan (PET-CT)
Observe the change in FDG uptake in the lesions of the subjects compared to the baseline, assessed every three months.
Response rate of critical organs
Evaluate the changes in clinical symptoms compared to the baseline according to CTCAE 5.0. The assessment of clinical efficacy is conducted every three months after KQ-2003 CAR T-cells transfusion therapy.
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