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Table 1 Summary of clinical trials targeting the bone marrow microenvironment in AML

From: Leukemia stem cell-bone marrow microenvironment interplay in acute myeloid leukemia development

Target Regimen ClinicalTrial.gov Identifier Patient population Phase Response
VEGF Bevacizumab + cytarabine/idarubicin NCT 00096148 Untreated, < 60 years II  
Bevacizumab + cytarabine/mitoxantrone hydrochloride NCT00015951 Relapsed/refractory, ≥ 18 years II CR 33%
RTK Sunitinib NCT 00783653 Untreated, FLT3-ITD, ≥ 60 years I/II CR + CRi 59%
Tubulin Combretastatin A1 + cytarabine NCT02576301 Relapsed/refractory, ≥ 18 years I/II CR + CRi 15%
CXCR4 Plerixafor + decitabine NCT 01352650 Untreated, ≥ 60 years I  
Plerixafor + cytarabine/daunorubicin NCT 00990054 Untreated, 18–70 years I CR 67%
Plerixafor + sorafenib/G-CSF NCT 00943943 Relapsed/refractory, FLT3-ITD, ≥ 18 years I CR + CRi 36%
Plerixafor + mitoxantrone/etoposide/cytarabine (MEC) NCT 00512252 Relapsed/refractory, 18–70 years I/II CR + CRi 46%
Plerixafor + G-CSF/mitoxantrone/etoposide/cytarabine (MEC) NCT 00906945 Relapsed/refractory, 18–70 years I/II CR + CRi 30%
Plerixafor + cytarabine/etoposide NCT 01319864 Relapsed/refractory, 3–29 years I/II  
Plerixafor + G-CSF/ busulfan/fludarabine/thymoglobulin NCT 00822770 Allo-SCT, 18–65 years I/II  
Plerixafor + daunorubicin/clofarabine or daunorubicin/cytarabine NCT 01236144 Untreated, ≥ 60 years I/II  
Plerixafor + clofarabine NCT 01160354 Untreated, ≥ 60 years I/II  
Plerixafor + fludarabine/idarubicin/cytarabine/G-CSF (FLAG) NCT 01435343 Relapsed/refractory, 18–65 years I/II  
BL-8040 + cytarabine NCT 01838395 Relapsed/refractory, 18–75 years II CR + CRi 39%
BL-8040 + atezolizumab NCT 03154827 Relapsed/refractory, ≥ 60 years Ib/II  
Ulocuplumab NCT 01120457 Relapsed/refractory, ≥ 18 years I CR + CRi 51%
CXCL12 CX-01 + cytarabine/idarubicin NCT 02056782 Untreated, ≥ 60 years II CR 92%
CX-01 + cytarabine/idarubicin NCT 02873338 Untreated, ≥ 60 years II CR + CRi 89%
CX-01 + azacytidine NCT 02995655 Relapsed/refractory, ≥ 18 years I  
E-Selectin GMI-1271 + idarubicin/mitoxantrone/etoposide/cytarabine (MEC) NCT 02306291 Relapsed/refractory, or untreated, ≥ 60 years I/II  
GMI-1271 + mitoxantrone/etoposide/cytarabine (MEC) or fludarabine/cytarabine/idarubicin (FAI) NCT 03616470 Relapsed/refractory, 18–75 years III  
GMI-1271 + daunorubicin/cytarabine NCT 03701308 Untreated, ≥ 60 years II/III  
VLA-4 AS101 + chemotherapy NCT 01010373 Untreated, ≥ 60 years II  
Hypoxia TH-302 NCT 01149915 Relapsed/refractory, ≥ 18 years I CR + CRi 5%
PR-104 NCT 01037556 Relapsed/refractory, ≥ 18 years I/II CR + CRp 32%
  1. AML acute myeloid leukemia, VEGF vascular endothelial growth factor, RTK receptor tyrosine kinase, CR complete remission, CRi complete remission with incomplete count recovery, CRp complete remission with incomplete platelet count recovery, G-CSF granulocyte-colony stimulating factor, Allo-SCT allogeneic stem cell transplantation