Master protocol for cell therapy, Phase 1 proof-of-concept studies in relapsed and refractory multiple myeloma and includes long-term safety follow-up.
This trial is currently open and accepting patients.
Enrollment: 65 patients (estimated)View More
November 15, 2022
At the last data-cut performed on May 3, 2022, 37 patients (pts, median age 66 (range: 44-76)) were enrolled, 33 received CART-ddBCMA and 31 were evaluable for initial safety and clinical response. Median follow-up after CART-ddBCMA infusion was 12.1 months. 27 subjects received DL1 (6 in dose escalation and 21 in dose expansion, 25 evaluable) and 6 received DL2 (all evaluable). CAR+ cells comprised median 72% (48-87%) of total CD3+ T cells; median VCN was 2.2 copies/cell (1.1-3.5); median cell viability was 99% (91-100%), and median cell manufacturing yield was 1159 million cells (470-1626).
Subjects had a median of 5 (3-16) prior lines of therapy. Twenty-four pts (77%) were "triple-refractory," and 21 (68%) were "penta-refractory"; 12 pts (39%) had high tumor burden with ≥ 50% bone marrow plasma cells; and 12 (39%) pts (50%) had extramedullary disease at baseline. CRS was seen in 28/31 (90%) pts but only 1 subject (in DL2) had grade (G) 3 CRS and all other cases were G≤2. ICANS was seen in 7 subjects (5, G≤2; 2, G3), with 1 G3 case in each DL. All cases resolved without sequalae with standard management. No cases of off-tumor cell mediated toxicity, no delayed neurotoxicity events (i.e., occurring after day 28), and no Parkinsonian-like symptoms.
All 31 evaluable subjects demonstrated clinical response per IMWG criteria (ORR, 100%) with 22 sCR/CR (≥CR rate, 71%), 7 VGPR (≥VGPR rate, 94%), and 2 PR. Responses deepened over time and conversion to CR/sCR was observed with longer follow-up (as late as month 12). Of those evaluable for MRD testing to date (n=22), 19 (86%) were MRD-neg at 10-5 or lower.
Median duration of response, PFS & OS were not evaluable at the time of data-cut because 22 of 31 evaluable subjects (71%) had ongoing response. In the patients with ≥12 months follow-up (n=16), which included 8 (50%) patients with EMD, ORR was 100%, ≥CR rate was 81% (13/16) and ≥VGPR rate was 88% (14/16). CART-ddBCMA product characteristics were consistent with the specifications in all the lots and there were no manufacturing failures.
June 03, 2022
Evaluable for efficacy and safety analysis were 31 patients, based on follow-up of at least one month, following treatment. These evaluable patients comprised the dose escalation cohorts for the first dose level (100 million CAR+ T cells, n=6), the second dose level (300 million CAR+T cells, n=6), and a dose expansion cohort at the recommended Phase 2 dose (RP2D) of 100 million CAR+T cells (n=19). All patients enrolled in the study have poor prognostic factors with 21 of 31 (68%) patients penta-refractory, 12 of 31 (39%) extramedullary disease (EMD), and all 31 patients having had at least three prior treatments.
The interim CART-ddBCMA clinical results (May 3, 2022 cutoff date) demonstrate deep and durable responses in patients with poor prognostic factors.
June 04, 2021
As previously announced, as of the April 14, 2021 data cutoff:
All patients enrolled in the study have poor prognostic factors with 10 of 12 patients penta-refractory and all 12 patients having had at least three prior treatments.
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May 19, 2022
February 28, 2020
October 04, 2019
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