A Phase II study of HBI0101 (NXC-201) BCMA-CART in Multiple Myeloma and Light-chain Amyloidosis Patients. The goal of the study is to evaluate the efficacy and safety of HBI0101 CART.
Up to 180 subjects with relapsed/refractory (R/R) multiple myeloma (MM) or light chain amyloidosis (AL) will be enrolled in a single-arm, open-label, single-site Phase 2 study. Eligible subjects will undergo leukapheresis procedure to provide starting material for manufacturing of HBI0101 CART investigational product. Each eligible subject will receive a single dose of HBI0101 CART cells. Prior to administration of HBI0101 CART, the study subjects will undergo lymphodepletion. Following administration of HBI0101 CART the subjects will be hospitalized for several days and then will return for routine follow-up periodical visits until 24 months after infusion.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
180
HBI0101 CART is defined as autologous T cells transduced ex-vivo with anti-BCMA CAR retroviral vector encoding the chimeric antigen receptor (CAR) targeted to human BCMA. The HBI0101 CART may be provided fresh or cryopreserved.
Hadassah MO
Jerusalem, Israel
RECRUITINGClinical response to HBI0101 CART
Percentage of subjects who achieved partial response (PR) or better
Time frame: 24 months
Safety of HBI0101 CART
Incidence of Serious Adverse Events and Adverse Events of Special Interest related to study treatment
Time frame: 24 months
Overall response rate
Overall Response Rate is defined by proportion of subjects with Partial Response or better per International Myeloma Working Group (IMWG) Criteria for MM, and Partial Response or better per Hematologic Response Criteria for AL.
Time frame: 24 months
Overall survival
Overall Survival is defined by the date of the initial infusion of HBI010 CART cells to the date of the patient's death due to any cause. If the patient is alive or the vital status is unknown, then their data will be censored at the date they were last known to be alive.
Time frame: 24 months
Progression-free survival
Progression-Free Survival is defined as the time from the date of the initial infusion of HBI0101 CART to the date of first documented disease progression, or death due to any cause, whichever occurs first. This is defined as Stable Disease (or better) per IMWG Criteria for MM, and Partial Response or better per Hematologic Response Criteria for AL.
Time frame: 24 months
Duration of response
Duration of response is defined as the first observation of partial response (for patients undergoing bridging therapy, this may be the date of the initial CAR T-cell infusion), to the time of disease progression, with deaths from causes other than progression censored.
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Time frame: 24 months
Disease-free survival
Disease-Free Survival proportion of MRD evaluable subjects who are MRD negative is defined as the duration from start of complete response until the time of relapse from complete response. Minimal Residual Response (MRD) is defined as the absence of clonal plasma cells on bone marrow aspirate (minimum test sensitivity to detect 1 in 105 nucleated cells (10-5 threshold)).
Time frame: 24 months
Persistence of HBI0101 CART cells
Quantification of HBI0101 CART cells in the blood over time
Time frame: 24 months
Organ response
Frequency of organ response (including cardiac, renal, and hepatic responses) in subject with Light-chain Amyloidosis
Time frame: 24 months