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