Table 1.
Major prospective trials of CD19-targeting CAR T-cells.
| Reference | Costimulatory Domain | Study Population and Median Age | Design | N | CR (%) MRD-CR (%) |
Survival | CRS (%) Grade ≥ 3 (%) |
ICANS (%) Grade ≥ 3 (%) |
|---|---|---|---|---|---|---|---|---|
| NCT01593693 [24,26] | CD28 | Pediatric and young adults 13.5 y |
Phase 1 | 50 | 62% 56% |
Median OS: 10.5 m | 70% 22% |
20% 8% |
| NCT01865617 [27] | 4-1 BB | Adults 39 y |
Phase 1–2 | 53 | 85% 85% |
NA | 75% 19% |
23% 23% |
| NCT01044069 [25] | CD28 | Adults 42 y |
Phase 1 | 53 | 83% 67% |
Median OS: 12.9 m Median EFS: 6.1 m |
85% 26% |
44% 42% |
| NCT01626495 and NCT01029366 [28] | 4-1BB | Pediatric and adult 14 y |
Phase 1–2 | 30 | 90% 73% |
6-month OS: 78% 6-month EFS: 67% |
100% 27% |
43% |
| NCT02435849 (ELIANA) [19] | 4-1 BB | Pediatric and young adult 11 y |
Phase 2 | 75 | 81% 81% |
12-month OS: 76% 12-month EFS: 50% |
77% 46% |
40% 13% |
| NCT02614066 (ZUMA-3) [20] | CD28 | Adult patients 40 y |
Phase 2 | 55 | 71% 71% |
Median OS: 18.2 m Median RFS: 11.6 m |
89% 24% |
60% 25% |
| NCT02028455 (PLAT-02) [21] | 4-1 BB | Pediatric and young adult 12.3 y |
Phase 1–2 | 43 | 93% | 12-month OS: 69.5% 12-month EFS: 50.8% |
93% 23% |
49% 21% |
| NCT01860937 [29] | CD28 | Pediatric and young adult 13.5 y |
Phase 1 | 25 | 75% | NA | 80% 16% |
72% 28% |
| NCT02030847 and NCT01029366 [30] | 4-1 BB | Adults 33.8 y |
Phase 1–2 | 35 | 69% | Median OS: 19.1 m Median EFS: 5.6 m |
94% 72% |
43% 6% |
| NCT02735291 [31] | 4-1 BB | Pediatric and adults 22 y |
Phase 2 | 47 | 81% 79% |
12-month OS: 53% 12-month RFS: 45% |
83% 23.4% |
4.3% 2.1% |
| NCT02935257 (ALLCAR19) [32] | 4-1 BB | Adults 41.5 y |
Phase 1 | 20 | 85% | 12-month OS: 64% 12-month EFS: 48% |
55% 0% |
20% 15% |
N: number; CR: complete remission; MRD-: minimal residual disease negative; CRS: cytokine release syndrome; ICANS: immune effector cell-associated neurologic syndrome; OS: overall survival; EFS: event-free survival; RFS: relapse-free survival; NA: not available.