Table 7 Ongoing clinical trials of checkpoint inhibitors in AML.
From: T-cell-based immunotherapy of acute myeloid leukemia: current concepts and future developments
Setting | Checkpoint target | Intervention | Study design | Study population | Status | NCT.gov identifier |
|---|---|---|---|---|---|---|
Single agent | PD-1 and CTLA-4 | Nivolumab, ipilimumab | Phase I Three cohorts • Nivolumab • Ipilimumab • Nivolumab + ipilimumab | High-risk or R/R AML and MDS following allo-HSCT | Recruiting | NCT03600155 |
PD-1 CTLA-4 | Nivolumab, ipilimumab | Phase I Three cohorts • Nivolumab ipilimumab • Nivolumab + ipilimumab | AML and MDS at high risk of relapse post-allo-HSCT | Recruiting | NCT02846376 | |
PD-1 CTLA-4 | Nivolumab, ipilimumab | Phase I Single cohort • Nivolumab or ipilimumab | Relapsed hematologic malignancies (including AML) post-allo-HSCT | Active, not recruiting | NCT01822509 | |
PD-1 | Nivolumab | Phase I Two cohorts • Nivolumab post-HLA-matched unrelated donor HSCT • Nivolumab post-HLA-haploidentical donor HSCT | High-risk patients with MDS and AML post-allo-HSCT with post-transplant cyclophosphamide | Recruiting | NCT04361058 | |
PD-1 | Nivolumab | Phase II Single cohort • Nivolumab | AML in remission at high risk for relapse | Recruiting | NCT02532231 | |
PD-1 | Nivolumab | Randomized phase II Two cohorts • Nivolumab observation | AML patients in first complete remission after chemotherapy | Active, not recruiting | NCT02275533 | |
PD-1 | Pembrolizumab | Phase I Single cohort • Pembrolizumab | AML, ALL or MDS with post-transplant relapse | Recruiting | NCT03286114 | |
PD-1 | Pembrolizumab | Pilot study Single cohort • Pembrolizumab | AML, MDS, or mature B-cell lymphomas that have relapsed following allo-HSCT | Recruiting | NCT02981914 | |
PD-1 | Pembrolizumab | Phase II Single cohort • Pembrolizumab | AML in remission not eligible for allo-HSCT | Active, not recruiting | NCT02708641 | |
Combination with HMA | CTLA-4 | Ipilimumab, decitabine | Phase I Two cohorts • Decitabine + ipilimumab for relapse post-allo-HSCT • Decitabine + ipilimumab for relapse in transplant-naïve patient | R/R AML or MDS | Recruiting | NCT02890329 |
PD-1 CTLA-4 | Nivolumab, ipilimumab, azacitidine | Phase II, nonrandomized Two cohorts • Azacitidine + nivolumab • Azacitidine + nivolumab + ipilimumab | Patients with R/R or newly diagnosed AML | Recruiting | NCT02397720 | |
PD-1 | Pembrolizumab, decitabine | Phase I Two cohorts • Pembrolizumab + decitabine for patients with AML • Pembrolizumab + decitabine for patients with MDS | Patients with R/R or newly diagnosed AML or MDS | Not yet recruiting | NCT03969446 | |
PD-1 | Nivolumab, azacitidine | Phase I Single cohort • Azacitidine + nivolumab post-HSCT | AML and high-risk myelodysplasia following reduce intensity allogeneic PBSC | Recruiting | NCT04128020 | |
PDL-1 | Durvalumab, azacitidine | Randomized phase II Two cohorts • Azacitidine + durvalumab • Azacitidine alone | Higher-risk MDS or in elderly (≥65 years) AML subjects not eligible for HSCT | Active, not recruiting | NCT02775903 | |
Combination with cytotoxic chemotherapy | PD-1 | Pembrolizumab, conventional intensive chemotherapy | Randomized phase II Two cohorts • Conventional intensive chemotherapy + pembrolizumab • Conventional intensive chemotherapy | Newly diagnosed AML eligible for intensive induction chemotherapy | Not yet recruiting | NCT04214249 |
PD-1 | Nivolumab, idarubicin, cytarabine | Phase II • Single cohort • Idarubicin + cytarabine + nivolumab | High-risk AML and MDS | Active, not recruiting | NCT02464657 | |
Other combination | PD-1 | Ivosidenib (AG-120), nivolumab | Phase II Single cohort • Ivosidenib + nivolumab | Patients with IDH1-mutated R/R AML and high-risk MDS | Recruiting | NCT04044209 |
PD-1 | Pembrolizumab, azacitidine, venetoclax | Randomized phase II Two cohorts • Azacitidine + VEN • Azacitidine + VEN + pembrolizumab | Older patients with AML who are ineligible or refuse intensive chemotherapy | Not yet recruiting | NCT04284787 | |
PD-1 | Nivolumab, decitabine, venetoclax | Phase I Single cohort • Nivolumab + decitabine + venetoclax | TP53-mutated AML | Recruiting | NCT04277442 | |
PDL-1 | Atezolizumab, gilteritinib | Phase I/II Single cohort • Atezolizumab + gilteritinib | R/R FLT3-mutated AML | Active, not recruiting | NCT03730012 | |
PD-1 | PDR001, decitabine MBG453 | Randomized phase I Five cohorts • Decitabine + PDR001 • Decitabine + MBG453 • Decitabine + PDR001 + MBG453 • MBG453 + PDR001 • MBG453 alone | Patients with R/R AML or high-risk MDSD | Recruiting | NCT03066648 | |
CTLA-4 | Ipilimumab CD25/Treg-depleted DLI | Phase I Single cohort • CD25/Treg-depleted DLI + ipilimumab | Myeloid disease relapse after matched HSCT | Recruiting | NCT03912064 | |
PDL- 1 | Atezolizumab Hu5F9-G4 | Phase I Single cohort • Atezolizumab + Hu5F9-G4 | R/R FLT3-mutated AML | Recruiting | NCT03922477 |
