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Table 1 Multiple myeloma CAR-T-cell clinical trials targeting BCMA

From: Safety and clinical efficacy of BCMA CAR-T-cell therapy in multiple myeloma

Trial # ref. (product name) n =  Origin mAb Expansion Loading Co-stimulation T-cell dosage Conditioning Toxicity Clinical response
ChiCTR-OIC17011272 [27] (CD19 & BCMA CAR-T) 21 Murine scFv aCD3 Lentiviral 4-1BB 1 × 106/kg CP/Flu CRS gr. 1–2 (86%), gr. ≥ 3 (5%)
Neurotoxicity (10%)
sCR/CR (57%)/VGPR (24%)
PR (14%)
NCT02658929 [24, 28] (bb2121) 43/39 Murine scFv aCD3 + aCD28 Lentiviral 4-1BB 50–800 × 106 CP/Flu CRS gr. 1–2 (58%), gr. ≥ 3 (5%)
Neurotoxicity (33%)
sCR/CR (44%)/VGPR (23%)
PR (10%)
NCT03274219 [29] (bb21217) 38 Murine scFv aCD3 + aCD28
 + PI3k inhibitor
Lentiviral 4-1BB 150–450 × 106 CP/Flu CRS gr. 1–2 (61%), gr. ≥ 3 (5%)
Neurotoxicity (24%)
sCR/CR (13%)/VGPR (34%)
PR (5%)
ChiCTR-OPC16009113 [30, 31] (BCMA-CAR T) 28 Murine scFv aCD3 Lentiviral CD28/4-1BB 5.4–25 × 106/kg CP/Flu CRS gr. ≥ 3 (14%) sCR/CR (61%)/VGPR (4%)
PR (21%)
NCT02215967 (1) [25, 32] (NCI BCMA CAR-T) 10 Murine scFv aCD3
+ IL-2
Retroviral CD28 0.3–3 × 106/kg CP/Flu CRS gr. 1–2 (30%) VGPR (10%)
PR (10%)
NCT02215967 (2) [25] (NCI BCMA CAR-T) 16 Murine scFv aCD3
+ IL-2
Retroviral CD28 9 × 106/kg CP/Flu CRS gr. 1–2 (56%), gr. ≥ 3 (38%)
Neurotoxicity (6%)
sCR/CR (13%)/VGPR (50%)
PR (19%)
ChiCTR-1800018143 [33] (BM38 CAR) 22 Humanized scFv ND ND 4-1BB 0.5–4 × 106/kg CP/Flu CRS gr. 1–2 (68%), gr. ≥ 3 (23%) sCR/CR (55%)/VGPR (9%)
PR (24%)
NCT02546167 [20] (CART-BCMA UPenn) 25 Human scFv aCD3/CD28 Lentiviral 4-1BB 50–500 × 106 CP or none CRS gr. 1–2 (56%), gr. ≥ 3 (32%)
Neurotoxicity (32%)
sCR/CR (8%)/VGPR (20%)
PR (20%)
NCT03302403, NCT03380039, NCT03716856 [34, 35] (CT053) 24 Human scFv aCD3/CD28 Lentiviral 4-1BB 50–180 × 106 CP/Flu CRS gr. 1–2 (63%)
Neurotoxicity (8%)
sCR/CR (79%)/VGPR (4%)
PR (4%)
NCT03430011 [36] (JCARH125) 44 Human scFv ND Lentiviral 4-1BB 50–450 × 106 CP/Flu CRS gr. 1–2 (70%), gr. ≥ 3 (9%)
Neurotoxicity (25%)
sCR/CR (27%)/VGPR (20%)
PR (34%)
NCT03815383 [37] (C-CAR088) 5 Human scFv ND Lentiviral 4-1BB 1–3 × 106/kg CP/Flu CRS gr. 1–2 (80%) sCR/CR (20%)/VGPR (60%)
PR (20%)
ChiCTR-1800018137 [38] (CT103A) 18 Human scFv ND Lentiviral 4-1BB 1–6 × 106/kg CP/Flu CRS gr. 1–2 (72%), gr. ≥ 3 (22%) sCR/CR (67%)/VGPR (17%)
PR (17%)
NCT03549442 [39] (CART-BCMA + CTL119) 16 Human scFv ND Lentiviral 4-1BB 500 × 106 CP/Flu CRS gr. 1–2 (88%) sCR/CR (19%)/VGPR (25%)
PR (25%)
NCT03338972 [40] (FCARH143) 11 Human scFv aCD3/CD28 Lentiviral 4-1BB
+ EGFRt 
50–150 × 106 CP/Flu CRS gr. 1–2 (91%)
Neurotoxicity (9%)
sCR/CR (55%)/VGPR (36%)
PR (9%)
NCT03502577 [41] (FCARH143 + GSI) 10 Human scFv ND Lentiviral 4-1BB
+ EGFRt 
50–300 × 106 CP/Flu CRS gr. 1–2 (60%), gr. ≥ 3 (40%)
Neurotoxicity (60%)
sCR/CR (30%)/VGPR (50%)
PR (20%)
NCT03196414 [42] (SZ-MM-CART01) 29/28 Humanized scFv aCD3 Lentiviral CD28/OX40 20–82 × 106/kg CP/Flu CRS gr. 1–2 (66%), gr. ≥ 3 (34%)
Neurotoxicity (3%)
sCR/CR (54%)/VGPR (4%)
PR (29%)
NCT03455972 [43] (SZ-MM-CART02) 32 Humanized scFv aCD3 Lentiviral CD28/OX40 50 × 106/kg BUCY or Mel
+ autoHSCT 
CRS gr. 1–2 (97%), gr. ≥ 3 (3%) sCR/CR (72%)/VGPR (ND)
PR (ND)
NCT03070327 [26] (MCARH171) 10/11 Human scFv ND Retroviral 4-1BB
 + EGFRt
1 × 106/kg or
150–450 × 106
CP/Flu or CP CRS gr. 1–2 (40%), gr. ≥ 3 (20%)
Neurotoxicity (10%)
VGPR (45%)
PR (18%)
NCT03602612 [44] (FHVH33) 15 Human VH ND Retroviral 4-1BB ND CP/Flu CRS gr. 1–2 (87%), gr. ≥ 3 (7%)
Neurotoxicity (27%)
sCR/CR (20%)/VGPR (7%)
PR (53%)
NCT03288493 [45] (P-BCMA-101) 23/19 Human centyrin None Transposon 4-1BB
 + rimiducid SS
51–1143 × 106 CP/Flu CRS gr. 1–2 (9%)
Neurotoxicity (4%)
sCR/CR + VGPR (26%)
PR (42%)
NCT03661554 [46] (BCMA nanoantibody) 9 Alpaca VHH ND Lentiviral 4-1BB 250–900 × 106 CP/Flu CRS gr. 1–2 (67%), gr. ≥ 3 (22%)
Neurotoxicity (11%)
sCR/CR (56%)/VGPR (33%)
PR (11%)
NCT03090659 (1) [47, 48] (LCAR-B38M) 17 Llama VHH aCD3/CD28
+ IL-2
Lentiviral 4-1BB 0.21–1,52 × 106/kg CP/Flu or CP CRS gr. 1–2 (59%), gr. ≥ 3 (41%) sCR/CR (82%)/VGPR (6%)
NCT03090659 (2) [49, 50] (LCAR-B38M) 57 Llama VHH aCD3/CD28
+ IL-2
Lentiviral 4-1BB 0.07–2,1 × 106/kg CP CRS gr. 1–2 (82%), gr. ≥ 3 (7%)
Neurotoxicity (2%)
sCR/CR (73%)/VGPR (4%)
PR (11%)
NCT03548207 [51] (LCAR-B38M) 29 Llama VHH ND Lentiviral 4-1BB 0.5–0.9 × 106/kg CP/Flu CRS gr. 1–2 (86%), gr. ≥ 3 (7%)
Neurotoxicity (10%)
sCR/CR (69%)/VGPR (17%)
PR (14%)
ChiCTR-1800017404 [52] (BCMA CAR-T) 33/32 ND ND Lentiviral 4-1BB 1–6 × 106/kg CP/Flu CRS gr. 1–2 (52%), gr. ≥ 3 (48%) sCR/CR (66%)/VGPR (22%)
PR (13%)
NCT03093168 [53] (HRAIN BCMA-CART) 49 ND ND Retroviral 4-1BB
 + EGFRt
9 × 106/kg CP/Flu CRS gr. 1–2 (12%), gr. ≥ 3 (6%) sCR/CR (45%)/VGPR (18%)
PR (14%)
ChiCTR-1900027678 [54] (GC012F) 5 ND ND ND ND 1–2 × 106/kg CP/Flu or none CRS gr. 1–2 (80%) sCR/CR (20%)/VGPR (80%)
Pooled studies 639/630        CRS gr. 1–4 (80.3%) ORR (80.5%)
         (95% CI 69.0–88.2; I2= 83%) (95% CI 73.5–85.9; I2 = 61%)
         Neurotoxicity (10.5%)  
         (95% CI 6.8–16.0; I2= 58%)  
  1. aCD3 + aCD28 = anti-CD3 and anti-CD28 antibodies. aCD3/CD28 + IL-2 = anti-CD3 and anti-CD28-coated beads plus interleukin-2. AutoHSCT = autologous hematopoietic stem cell transplant. BCMA = B-cell maturation antigen. BUCY = busulfan and cyclophosphamide. CAR = chimeric antigen receptor. CP = cyclophosphamide. CR = complete response. CRS = cytokine release syndrome. EGFRt = truncated epidermal growth factor receptor. Flu = fludarabine. Gr. = grade. GSI = gamma-secretase inhibitor. IL-2 = interleukin-2. Mel = melphalan. n = number of patients evaluable for toxicity/clinical response. ND = not disclosed. PI3k = phosphoinositide 3-kinase. PR = partial response. scFv = single-chain fragment variable. SS = safety switch. sCR = stringent complete response. Trial # = study registration number in Clinicaltrials.gov (NCT#) or Chinese Clinical Trial Registry (ChiCTR-#). VGPR = very good partial response. VHH = nanobody