Table 1.
Ongoing clinical trials of immune checkpoint blockade in AML.
| Setting | Target | Drug | NCT number | Phase of study | Study population | Therapy regimen | Objective | Status |
|---|---|---|---|---|---|---|---|---|
| AML and high-risk MDS | PD-1 | Nivolumab | 02464657 | Phase I/II | AML or high-risk MDS | Nivolumab + idarubicin + cytarabine, single arm | MTD, EFS | Recruiting |
| Remission maintenance | PD-1 | Nivolumab | 02275533 | Phase II | AML in remission | Single agent, two arms | PFS; OS, toxities | Recruiting |
| PD-1 | Nivolumab | 02532231 | Phase II | AML in remission, high risk for relapse | Single agent, single arm | RFS (time frame 6 months) | Recruiting | |
| PD-L1 | Atezolizumab | 03154827 | Phase Ib/II | AML (>60 years) in remission | Atezolizumab + BL-8040 | RFS (time frame: up to 5 years) | Recruiting | |
| R/R AML | PD-1 | Nivolumab | 02397720 | Phase II | R/R AML or elderly de novo AML patients | Azacitidine+nivolumab or azacitidine+nivolumab+ipilimumab, two arms | MTD, ORR of nivolumab with azacitidine, adverse event | Recruiting |
| PD-1 | Pembrolizumab | 02768792 | Phase II | R/R AML | Pembrolizumab after high-dose cytarabine as induction therapy | OR (CR/Cri); toxicities | Recruiting | |
| PD-1 | Pembrolizumab | 02845297 | Phase II | R/R AML or elderly de novo AML patients | Pembrolizumab following azacitidine, single arm | MTD; ORR (CR, CRi) | Recruiting | |
| PD-1 | Pembrolizumab | 02996474 | Phase I/II | R/R AML | Pembrolizumab and decitabine | Feasibility; efficacy | Active, not recruiting | |
| PD-1 | Pembrolizumab | 03291353 | Early phase I | Refractory AML | Single agent, single arm | Adverse event; RR, OS | Recruiting | |
| PD-1/TIM-3 | PDR001/MBG453 | 03066648 | Phase I | R/R AML or de novo AML not suitable for standard therapy | Decitabine+PDR001 or decitabine+MBG453 or decitabine+PDR001+MBG453 or MBG453 alone or PDR001+MBG453 | Safety, DLT | Recruiting | |
| CTLA-4 | Ipilimumab | 01757639 | Phase I | R/R AML | Single agent, single arm | DLT, T-reg cell percentages; efficacy, PFS, OS | Completed | |
| CTLA-4 | Ipilimumab | 02890329 | Phase I | R/R AML or elderly de novo AML | Ipilimumab and decitabine | MTD; clinical response | Recruiting | |
| High risk or old age not eligible transplant | PD-1 | Pembrolizumab | 02708641 | Phase II | Elderly AML (>60 years) not eligible for transplantation | Single agent, single arm | Time to relapse; OS | Recruiting |
| PD-1 | Pembrolizumab | 02771197 | Phase II | High-risk AML not eligible for transplant | Pembrolizumab following lymphodepletion therapy (fludarabine+melphalan), single arm | 2-year relapse risk; safety | Recruiting | |
| PD-L1 | Durvalumab | 02775903 | Phase II | Elderly AML (>=65 years) not eligible for transplantation | Durvalumab+azacitidine | ORR (CR/CRi) | Active, not recruiting | |
| CTLA-4 | Ipilimumab | 00039091 | Phase I | AML in remission, not eligible for transplant | Single agent, single arm | Toxicities; ORR | Terminated | |
| Post transplant | PD-1 | Pembrolizumab | 03286114 | Phase I | AML relapse after allo-SCT | Single agent, single arm | Clinical benefit; response rate | Recruiting |
| PD-1 | Pembrolizumab | 02981914 | Early phase I | AML relapse after allo-SCT | Single agent, single arm | Adverse event; duration of response | Recruiting | |
| PD-1 | Ipilimumab+ nivolumab |
02846376 | Phase I | AML after allo-SCT | Ipilimumab or nivolumab or ipilimumab+nivolumab |
Safety (DLT); toxicities | Recruiting | |
| PD-1 | Ipilimumab or nivolumab | 01822509 | Phase I/Ib | AML relapse after allo-SCT | Ipilimumab or nivolumumab, single arm | MTD, adverse events; RR, PFS, OS | Active, not recruiting | |
| PD-1 | Ipilimumab or nivolumab | 03600155 | Phase I | High-risk AML or relapsed AML after allo-SCT | Nivolumab or ipilimumab or nivolumab+ipilimumab | MTD, DLT; ORR, DFS, OS | Recruiting | |
| CTLA-4 | Ipilimumab | 00060372 | Phase I | AML after allo-SCT | Single agent, single arm | Safety dose | Completed | |
| CTLA-4 | Ipilimumab | 01919619 | Early phase I | AML after allo/auto-SCT | Ipilimumab+lenalidomide | Toxicity rate (time frame: 28 days) | Recruiting |
AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; MTD, maximum tolerated dose; EFS, event-free survival; PFS, progression-free survival; OS, overall survival; RFS, recurrence-free survival; ORR, overall response rate; RR, response rate; DLT, dose limiting toxicity; DFS, disease-free survival; allo-SCT, allogeneic stem-cell transplantation; CR, complete remission; CRi, complete remission with incomplete hematologic recovery.
Objectives are listed as “primary; secondary” outcome measures.