This is a phase I/II, single-arm, open-lable study of autologous stem cell transplantation in combination with C-CAR088, an autologous BCMA CAR-T cell product, for patients with ulta high-risk multiple myeloma, defined as failed or unsatisfied responses to front line VRD-based treatment with or without the presence of multiple high-risk cytogenetic features.
Patients with ultra high-risk multiple myeloma will undergo leukapheresis, stem cell mobilization and collection (could omit if collected before screening), conditioning, ASCT and C-CAR088 infusion. Patients receive a single dose of C-CAR088 three days post-ASCT. Two conditioning protocols and two dose levels of C-CAR088 will be used based on the investigator's discretion. Patients will be evaluated closely for safety of efficacy during the first three months, then less frequently in the following months until 24 months post-ASCT.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Patients receive transplantation conditioning followed by autologous hematopoietic stem cell transplantation after successful stem cell mobilization and collection. If previously collected stem cells are available, no stem cell mobilization or collection is required, and patients will receive conditioning directly.
C-CAR088 is an BCMA targeted Chimeric Antigen Receptor-T cell product. Patients will receive C-CAR088 single dose infusion 3 days after ASCT. The dose level of C-CAR088 will be determined by the investigator.
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China
RECRUITINGIncidence rate and severity of adverse events (AE)
Incidence rate and severity of adverse events (AE)
Time frame: 24 months
Progression free survival (PFS)
The time from the initiation of study treatment to the date of first documented disease progression or death
Time frame: 24 months
MRD negativity rate
The percentage of patients who reached MRD negativity
Time frame: 24 months
Overall response rate (ORR)
The percentage of patients who reached PR, VGPR, CR or sCR as their best response
Time frame: 24 months
Duration of response (DOR)
The time from the first documented PR or better response to progression or death, whichever occurs first
Time frame: 24 months
Time to response (TTR)
The time between the initiation of study treatment until the the first documented PR or better response
Time frame: 24 months
Overall Survival (OS)
OS is defined as the time from the initiation of study treatment to death from any cause
Time frame: 24 months
Cmax (maximal plasma concentration)
Maximal plasma concentration of C-CAR088 in peripheral blood
Time frame: 24 months
Dehui Zou, M.D., PH.D.
CONTACT
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Tmax (Time to reach the maximal plasma conceration)
Time to reach the maximal plasma conceration of C-CAR088 in peripheral blood
Time frame: 24 months
AUC0-28d (area under the curve from day 0-day 28)
Area under the curve of C-CAR088 in peripheral blood within 28 days post C-CAR088 infusion
Time frame: 28 days post C-CAR088 infusion
Tlast (Time of last measurable observed concentration)
Time of last measurable observed concentration of C-CAR088 in peripheral blood
Time frame: 24 months