A Phase 1/2 Open label, multicenter, clinical trial of autologous CAR T-cell therapy targeting GPRC5D, in participants with relapsed/refractory multiple myeloma or relapsed/refractory primary plasma cell leukemia.
This is an open-label, multicenter, Phase 1/2 trial of CT071 in adult participants with relapsed or refractory multiple myeloma (RRMM) or relapsed or refractory primary plasma cell leukemia (RRpPCL). The study will be conducted in two phases. Phase 1 of the study will be dose escalation followed by dose expansion. After recommended Phase 2 dose is identified in Phase 1, the enrollment of Phase 2 will start. Following consent, enrolled subjects will undergo apheresis to collect cells for manufacture of the CAR-T cells. Following the manufacture of the CAR-T cells, subjects will receive lymphodepletion prior to CAR T-cell infusion. All subjects who complete the study, as well as those who withdraw from the study after receiving CAR T-cell infusion for reasons other than death or meeting the early termination criteria, will be asked to undergo a 15-year long-term follow-up.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
166
a single CAR-T infusion of CT071
Phase 1: Evaluation of the Safety of CT071 and determination of Maximum Tolerated Dose (MTD).
Frequency, type, and severity of AEs (SAEs, AESIs, laboratory abnormalities).
Time frame: Day 1 - Month 24
Phase 2: Objective response rate
Objective response rate (ORR) per IMWG by IRC read; percentage of participants achieving confirmed PR or better per IMWG 2016 consensus criteria.
Time frame: Day 1 - Month 24
Phase 1 and 2: Evaluate additional clinical efficacy outcomes
Overall Response Rate/Best Overall Response Rate by investigator (by IMWG stringent complete response/sCR, complete response/CR, very good partial response/VGPR, and partial response/PR by IRC and investigator assessment).
Time frame: Day 1 - Month 24
Phase 1 and 2: Evaluate additional clinical efficacy outcomes
Duration of Response by IMWG (stringent complete response/sCR, complete response/CR, very good partial response/VGPR, and partial response/PR by IRC and investigator assessment).
Time frame: Day 1 - Month 24
Phase 1 and 2: Evaluate additional clinical efficacy outcomes
Progression Free Survival by investigator assessment
Time frame: Day 1 - Month 24
Phase 1 and 2: Evaluate additional clinical efficacy outcomes
Overall Survival
Time frame: Day 1 - Month 24
Phase 2: Evaluate additional Safety of CT071.
Frequency, type, and severity of AEs (SAEs, AESIs, laboratory abnormalities).
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Time frame: Day 1 - Month 24
Phase 1 and 2: Assess immunogenicity of CT071
Percentage of patients with anti-CT071 drug antibodies
Time frame: Day 1 - Month 60
Phase 1 and 2: Evaluate PK profile of CT071
CAR transgene copy peak value
Time frame: Day 1 - Month 60
Phase 1 and 2: Evaluate PK profile of CT071
CAR transgene copy number persistence
Time frame: Day 1 - Month 60
Phase 1 and 2: Evaluate PK profile of CT071
CAR transgene copy AUC
Time frame: Day 1 - Month 60