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. Author manuscript; available in PMC: 2021 Aug 11.
Published in final edited form as: Blood Cancer Discov. 2021 Jul;2(4):302–318. doi: 10.1158/2643-3230.BCD-20-0227

Table 2.

Selected studies evaluating BCMA-specific CAR T-cells: efficacy and safety data.

NCI UPenn (Novartis) Ide-cel (BMS) bb21217 (BMS) LCAR-B38M Cilta-cel (Janssen) Orva-cel (JCARH125; BMS)
Stage of trial; Clinicaltrials.gov Identifier; reference Phase 1; NCT02215967(5) Phase 1; NCT02546167(9) Phase 1; NCT02658929 (CRB-401)(12) Phase 2; NCT03361748 (KarMMa)(10) Phase 1; NCT03274219 (CRB-402)(82) Phase1; NCT03090659 (LEGEND-2)(14) Phase 1b/2; NCT03548207 (CARTITUDE-1)(11) Phase 1/2; NCT0340011 (EVOLVE)(23)
≥PR 0.3–3×106/kg: 20%
9×106/kg: 81%		 48% 50–800×106: 76%
450×106 (n=38): 90%		 150–450×106: 73%
150×106 : 50%
300×106 : 69%
450×106 : 81%		 150–450×106: 68%
150×106 : 83%
300×106 : 43%
450×106 : 73%		 88% 97% 92%
≥CR 0.3–3×106/kg: 0%
9×106/kg: 13%		 8% 50–800×106: 39%
450×106 (n=38): 37%		 150–450×106: 33%
150×106 : 25%
300×106 : 29%
450×106 : 39%		 150–450×106: 29%
150×106 : 42%
300×106 : 14%
450×106 : 30%		 74% 67% 36%
MRD-negativity (assay used to assess MRD is also provided)

  • 100% of 11 evaluable patients treated with 9×106 CAR T-cells/kg and with ≥PR

  • NGF (0.0007% depth)

  • 57% of 7 patients with ≥VGPR

  • NGF (1×10−5 depth)

  • 100% of 15 evaluable patients with ≥CR

  • NGS (1×10−4 depth)

  • 75% of the 67 patients with ≥VGPR

  • 79% of the 42 patients with ≥CR

  • NGS (1×10−5 depth)

  • 89% of 28 evaluable patients with ≥PR

  • 100% of 13 evaluable patients with ≥CR

  • NGS (1×10−5 depth)

  • 93% of the 42 patients with CR

  • NGF (~10−4 depth)

  • 93% of 57 evaluable patients

  • NGS (1×10−5 depth)

  • 84% of 25 evaluable patients with ≥PR at month 3

  • NGS (1×10−5 depth)
Median PFS 9×106/kg: 31 weeks (EFS) Cohort 1: 65 days
Cohort 2: 57 days
Cohort 3: 125 days		 50–800×106 cells: 8.8 months
450×106 cells: 9.0 months		 150–450×106: 8.8 months
150×106 : 2.8 months
300×106 : 5.8 months
450×106 : 12.1 months		 PFS not reported; median duration of response: 17.0 months 19.9 months Not reached; 12-month PFS rate: 77% 300×106 : 9.3 months
450×106 :not reached
600×106 : not reached
Cytokine release syndrome (any grade)

  • 0.3–3×106/kg: 40%

  • 9×106/kg: 94%

 88% 76% 84% 65% 90% 95% 89%
Grade ≥3 cytokine release syndrome

  • 0.3–3×106/kg: 0%

  • 9×106/kg: 38%

 32% 7% 5% 4% 7% 4% 3%
Neurotoxicity (any grade) NR 32% 36% 18% 16% 2% 21%* 13%
Grade ≥3 neurotoxicity NR 12% 2% 3% 4% 0% 10%* 3%
*

Twelve out of 97 patients treated with cilta-cel in the CARTITUDE-1 study experienced neurotoxicity which occurred after resolution of cytokine release syndrome and/or immune effector cell-associated neurotoxicity syndrome (ICANS). Five patients experienced movement and/or neurocognitive changes, and 7 had adverse events including nerve palsy and peripheral motor neuropathy.

Abbreviations: NR, not reported; NGS, next-generation sequencing; NGF, next-generation flow cytometry; PR, partial response; VGPR, very good partial response; CR, complete response; sCR, stringent complete response.