Table 2 Ongoing clinical trials for TP53-mutated AML.

From: What have we learned about TP53-mutated acute myeloid leukemia?

Clinical Trial

Phase

Agent

Target/mechanism

Comments

NCT04582864

II

Flotetuzumab

Anti-CD3/CD123

Bispecific DART; studied for R/R AML and MDS after allo-HSCT

NCT03647800

Ib

APVO436

CD3/CD123

APVO436 + VEN + AZA for R/R AML or MDS

NCT03013998

I/II

Entospletinib

SYK

Entospletinib + DEC in newly diagnosed TP53 mutant AML

NCT05428969

I/II

Bexmarilimab

CLEVER-1

In combination with AZA or AZA/VEN in MDS, CMML, and AML

NCT04243499

I/II

ICT01

BTN3A/CD477

ICT01 + pembrolizumab in advanced stage R/R malignancies

NCT05396859

I

Entrecitinib

ROS-1

Entrecitinib + ASTX727 for R/R TP53 mutant AML. (ASTX727: DEC + cedazuridine)

NCT04477291

Ia/b

Luxeptinib (CG-806)

BTK

For RR AML/high-risk MDS

NCT05787496

I

NC525

LAIR-1

Open-label study for advanced myeloid neoplasms

NCT03855371

I

ATO

Multiple targets

ATO + DAC for TP53 mutant high-risk MDS/AML

NCT03560882

I

Atorvastatin

HMG Co-A reductase

Measuring change in conformational mutant TP53

NCT04266301

III

MBG453 (sabatolimab)

TIM-3

MBG453 in combination with AZA in intermediate-, high-, and very-high–risk MDS per IPSS-R, or CMML-2

NCT04277442

I

Nivolumab

PD-1

In combination with DEC and VEN in newly diagnosed TP53 mutated AML

NCT02890329

I

Ipilimumab

CTLA-4

Ipilimumab + DEC in R/R AML or MDS

NCT03018405

I

CYAD-01

NKG2D expressing CAR-T

THINK trial for multiple cancers

NCT01864902

I

CART33

CD33 CAR-T

For R/R CD33 positive AML

NCT02159495

I

MB-102

CD123 CAR-T

For R/R AML

NCT03795779

I

ICG-144

CLL1-CD33 CAR-T

In R/R high-risk hematologic malignancies

  1. allo-HSCT allogeneic hematopoietic stem cell transplant, AML acute myeloid leukemia, ATO arsenic trioxide, AZA azacitidine; CAR-T cell chimeric antigen receptor T cell, CMML, chronic monomyelocytic leukemia, DAC, daunorubicin, cytarabine, and cladribine, DART, dual affinity retargeting antibody, DEC, decitabine, IPSS-R, revised international prognostic scoring system, MDS, myelodysplastic syndrome, ROS-1, ROS proto-oncogene 1 receptor tyrosine kinase, R/R, relapse refractory, SYK, splenic tyrosine kinase, VEN, venetoclax.