Table 4.
Current clinical trials using checkpoint inhibitors for immunotherapy of AML
| Study identifier | Study name | Antigen/target | Drug name | Combination therapy | Clinical phase | Indication (AML only) | Primary endpoints | (Estimated) Enrollment |
Sponsor | Country | Study start | (Estimated) Completion date |
Status |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00039091 | Monoclonal antibody therapy in treating patients with ovarian epithelial cancer, melanoma, acute myeloid leukemia, myelodysplastic syndrome, or non-small cell lung cancer | CTLA-4 | Ipilimumab | n.a. | I | AML with different recurrent mutations or recurrent AML | Toxicity | 12 (AML only) | National Cancer Institute (NCI) | USA | 2002 | 2007 | Terminated |
| NCT01757639 | Ipilimumab in treating patients with relapsed or refractory high-risk myelodysplastic syndrome or acute myeloid leukemia | CTLA-4 | Ipilimumab | n.a. | I | Refractory AML | Toxicity, regulatory T cells | 54 (AML + MDS + CMML) | National Cancer Institute (NCI) | USA | 2012 | 2016 | Active, not recruiting |
| NCT02275533 | Nivolumab in eliminating minimal residual disease and preventing relapse in patients with acute myeloid leukemia in remission after chemotherapy | PD-1 | nivolumab | n.a. | II | AML in first remission; no eligibility for allo-HSCT | Clinical response (RFS) | 80 | National Cancer Institute (NCI) | USA | 2015 | 2019 | Recruiting |
| NCT02397720 | Study of Nivolumab (BMS-936558) in Combination With 5-azacytidine (Vidaza) for the Treatment of Patients With Refractory/ Relapsed Acute Myeloid Leukemia and Newly Diagnosed Older Acute Myeloid Leukemia (AML) (>65 Years) Patients | PD-1 | Nivolumab | Azacitidine | II | r/r AML or newly diagnosed older AML patients | MTD | 110 | M.D. Anderson Cancer Center | USA | 2015 | 2018 | Recruiting |
| NCT02464657 | Study of Idarubicin, Cytarabine, and Nivolumab in Patients With High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML) | PD-1 | Nivolumab | Idarubicin, cytarabine | I/II | De novo AML | MTD | 75 | M.D. Anderson Cancer Center | USA | 2015 | 2018 | Recruiting |
| NCT02532231 | Nivolumab in Acute Myeloid Leukemia (AML) in Remission at High Risk for Relapse | PD-1 | Nivolumab | n.a. | II | AML in remission with high risk of relapse | Clinical response (RFS) | 30 | M.D. Anderson Cancer Center | USA | 2015 | 2018 | Recruiting |
| NCT02708641 | A phase II study of pembrolizumab as post-remission treatment of patients ≥60 with AML | PD-1 | Pembrolizumab | n.a. | II | AML patients ≥60 years in CR; no eligibility for allo-HSCT | Toxicity, clinical response (time to relapse) | 40 | Alison Sehgal, MD, MS | USA | 2016 | 2021 | Not yet recruiting |
| NCT02768792 | High-dose cytarabine followed by pembrolizumab in relapsed/refractory AML | PD-1 | Pembrolizumab | High-dose cytarabine | II | r/r AML | Clinical response (CR rate) | 37 | UNC Lineberger Comprehensive Cancer Center | USA | 2016 | 2021 | Recruiting |
| NCT02771197 | Lymphodepletion and anti-PD-1 blockade to reduce relapse in AML patient not eligible for | PD-1 | Pembrolizumab | Fludarabine, melphalane, auto-SCT | II | Non-favorable risk AML in CR | Clinical response (2-y-RR) | 20 | Northside Hospital, Inc. | USA | 2016 | 2020 | Recruiting |
| NCT02775903 | An efficacy and safety study of azacitidine subcutaneous in combination with durvalumab (MEDI4736) in previously untreated subjects with higher-risk myelodysplastic syndromes (MDS) or in elderly subjects with acute myeloid leukemia (AML) | PD-L1 | Durvalumab | Azacitidine | II | De novo AML or sAML or tAML in elderly patients | Clinical response (RR) | 110 (AML alone) | Celgene Corporation | USA, Canada and various European countries | 2016 | 2019 | Recruiting |
| NCT02845297 | Phase 2 study of azacitidine in combination with pembrolizumab in relapsed/refractory acute myeloid leukemia (AML) patients and in newly diagnosed older (≥65 years) AML patients | PD-1 | Pembrolizumab | Azacitidine | II | r/r AML | MTD | 40 | Sidney Kimmel Comprehensive Cancer Center | USA | 2016 | 2020 | Recruiting |
| NCT02890329 | Ipilimumab and decitabine in treating patients with relapsed or refractory myelodysplastic syndrome or acute myeloid | CTLA-4 | Ipilimumab | Decitabine | I | r/r AML | MTD | 48 | National Cancer Institute (NCI) | USA | 2017 | 2019 | Not yet recruiting |
| NCT02890329 | Ipilimumab and decitabine in treating patients with relapsed or refractory myelodysplastic syndrome or acute myeloid | CTLA-4 | Ipilimumab | Decitabine | I | r/r AML or de novo AML in elderly patients | Toxicity, MTD | 48 (AML + MDS) | National Cancer Institute (NCI) | USA | 2017 | 2019 | Not yet recruiting |
| NCT02892318 | A study evaluating the safety and pharmacology of atezolizumab administered in combination with immunomodulatory agents in participants with acute myeloid leukemia (AML) | PD-L1 | Atezolizumab | Guadecitabine, possibly other immunomodulatory agents |
I | r/r AML or de novo AML in elderly patients | Toxicity, clinical response (CR, CRi, CRp, duration of response) | 40 | Hoffmann-La Roche | USA | 2016 | 2019 | Recruiting |
| NCT02953561 | Avelumab (antiPDL1) and azacytidine in acute myeloid leukemia (AML) | PD-L1 | Avelumab | Azacitidine | I/II | r/r AML | Toxicity | 52 | M.D. Anderson Cancer Center | USA | 2017 | 2020 | Not yet recruiting |
| NCT02996474 | Pembrolizumab and decitabine for refractory or relapsed acute myeloid leukemia | PD-1 | Pembrolizumab | Decitabine | I/II | r/r AML | Feasibility | 15 | National Heart, Lung, and Blood Institute (NHLBI) | USA | 2016 | 2019 | Not yet recruiting |