Table 1.
Characteristics, efficacy and safety data from selected clinical trials of BCMA-targeting CAR-T cell constructs.
| CAR-T Cell Construct (Name) | Study Name and/or Phase |
Number of Patients | Triple-Class Refractory, % | High-Risk Cytogenetics/EMD, % | Median PFS, Months (95% CI) | Median OS, Months (95% CI) |
≥CR, % | CRS, All Grades, % |
Neurotoxicity, All Grades, % | NCT Number | References |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Ide-cel (bb2121) | CRB-401, Phase 1 |
62 | 69 | 27/37 | 8.8 (5.9–11.9) | 34.2 (19.2–NE) | 39 | 76 | 36 | NCT02658929 | [18,19] |
| Ide-cel (bb2121) | KarMMa, Phase 2 |
128 | 84 | 35/39 | 8.8 (5.6–11.6) | 19.4 (18.2–NE) | 33 | 84 | 18 | NCT03361748 | [20,21] |
| Cilta-cel | LEGEND-2, Phase 1/2 |
57 | NR | NR | 20 (10–28) | Not reached, 18-month OS 68% (54–79%) | 74 | 90 | 1 | NCT03090659 | [26] |
| Cilta-cel | CARTITUDE-1, Phase 1b/2 |
97 | 88 | 24/13 | 22.8 (22.8–NE) | Not reached, 18-month OS 80.9% (71.4–87.6%) | 80 | 95 | 21 | NCT03548207 | [27,29] |
| Orva-cel | EVOLVE, Phase 1/2 |
62 | 94 | 41/23 | 9.3 in the 300 × 106 group (n = 19), not reached in the other groups | NR | 36 | 89 | 13 | NCT03430011 | [33] |
| bb21217 | CRB-402, Phase 1 |
69 | 64 | 33/NR | NR, mDOR 17.0 (9.4–NE) |
NR | 29 | 70 | 16 | NCT03274219 | [24] |
| NCI CAR-BCMA | Phase 1 | 24 | NR | 46/NR | NR, mEFS 31 weeks |
NR | 8 | 71 | NR | NCT02215967 | [13,34] |
| UPenn CART-BCMA | Phase 1 | 25 | 72 | 96/28 | 65/57/125 days in cohort 1/2/3 | NR | 8 | 88 | 32 | NCT02546167 | [35] |
| P-BCMA-101 | PRIME, Phase 1/2 |
55 | 60 | NR | NR | NR | NR, ≥VGPR: 50 (n = 6) * |
17 (n = 53) |
4 (n = 53) |
NCT03288493 | [32] |
| CT053 | LUMMICAR STUDY 2, Phase 1 |
20 | 85 | 55/25 | NR | NR | 25 | 79 | 16 | NCT03915184 | [36] |
| ALLO-715 | UNIVERSAL, Phase 1 |
31 | NR | 48/23 | NR | NR | ≥VGPR: 40 | 45 | 0 | NCT04093596 | [31] |
| C-CAR088 | Phase 1 | 23 | NR | 81/NR | Not reached, 6-month PFS 65.1% (47–90) |
NR | 44 | 91 | 4 |
NCT03751293 NCT03815383 NCT04322292 NCT04295018 |
[37] |
CAR = chimeric antigen receptor; CI = confidence interval; CR = complete remission; CRS = cytokine release syndrome; EMD = extramedullary disease; mDOR = median duration of response; mEFS = median event free survival; NE = not estimable: NR = not reported; OS = overall survival; PFS = progression free survival; VGPR = very good partial remission. * with modified manufacturing process. Only data of studies with results for at least 20 patients are reported.