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Table 4 Current clinical trials using checkpoint inhibitors for immunotherapy of AML

From: Recent developments in immunotherapy of acute myeloid leukemia

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