Cancer clinical trial database.
Every new trial and trial result from New in Oncology, our weekly research briefing, since the first issue on . Search by NCT number, drug, or cancer type.
All entries in acute myeloid leukemia and MDS
- Trial result
Acute myeloid leukemia and MDS
CPX-351 (liposomal daunorubicin/cytarabine) + CD33-targeted gemtuzumab ozogamicin delivers 19% CRc in heavily pretreated R/R AML
CPX-351, a fixed-ratio liposomal formulation of daunorubicin + cytarabine, was combined with gemtuzumab ozogamicin (GO), a CD33-directed calicheamicin antibody–drug conjugate, in a Phase 2 pilot study in…
NCT03672539 - New trial
Acute myeloid leukemia and MDS
Phase 1 ORM-1153 CD123-directed degrader-antibody conjugate delivers a GSPT1-degrading payload in hematologic malignancies
Orum Therapeutics USA is running this first-in-human, multicenter Phase 1 study of ORM-1153 in relapsed or refractory acute myeloid leukemia and other protocol-eligible CD123-expressing hematologic malignancies.…
NCT07809906 First-in-human - Trial result
Acute myeloid leukemia and MDS
CD123 CAR-T Delivers ~27% CR/CRi in Post-alloHCT AML With Manageable CRS/Neurotoxicity
Single-center, open-label Phase 1 dose-escalation evaluated CD123-directed CAR T cells (autologous or donor-derived allogeneic CAR-T targeting the AML/BPDCN antigen CD123) after lymphodepletion in CD123+…
NCT02159495 - New trial Spotlight
Acute myeloid leukemia and MDS
Phase 2/3 Pivekimab sunirine CD123-targeted ADC (IGN payload) + venetoclax/azacitidine in AML
AbbVie’s randomized Phase 2/3 trial (NCT07581002) tests whether adding pivekimab sunirine (PVEK)—a CD123-directed monoclonal antibody–drug conjugate carrying an indolinobenzodiazepine (IGN) DNA-alkylating payload—can…
NCT07581002 Registrational - New trial
Acute myeloid leukemia and MDS
Phase 1b monzosertib CDC7 kinase inhibitor tests azole–drug interaction dosing in AML/MDS
MD Anderson Cancer Center is running this Phase 1b, open-label, investigator-initiated study to define the recommended Phase 2 dose of monzosertib, a small-molecule CDC7 (cell-division cycle 7) kinase inhibitor that…
NCT07780565 Exploratory - New trial
Acute myeloid leukemia and MDS
Phase 1 arsenic trioxide p53-reactivating agent with fludarabine/busulfan allo-HSCT in AML/MDS
University of Michigan Rogel Cancer Center is running this Phase 1, non-randomized study testing whether adding arsenic trioxide (ATO)—a small molecule reported to help restore wild-type conformation/function of some…
NCT07737769 Exploratory - Trial result
Acute myeloid leukemia and MDS
Venetoclax (BCL-2 inhibitor) + azacitidine: 28-day induction trends to higher CR vs 14-day in OPTI-AML ND-AML
Venetoclax (Ven; oral BCL-2 inhibitor) plus azacitidine (Aza; hypomethylating agent) was tested in OPTI-AML (NCT03013998), a prospective randomized Phase 2 trial in genomically agnostic newly diagnosed AML patients…
NCT03013998 - Trial result
Acute myeloid leukemia and MDS
CPX-351 (liposomal daunorubicin/cytarabine) vs CLAG-M (high-dose cytarabine backbone) in unfit AML: higher CR/CRi with CLAG-M, no OS win overall
CPX-351 is a fixed-ratio liposomal formulation of daunorubicin + cytarabine, while CLAG-M combines cladribine + high-dose cytarabine + G-CSF + mitoxantrone (intensive cytarabine-based chemotherapy). In this…
NCT04195945 - Trial result
Acute myeloid leukemia and MDS
Venetoclax (BCL-2 inhibitor) + high-dose cytarabine drives 57% CR/CRi in pediatric R/R AML (VENAML)
VENAML tested venetoclax, an oral BCL-2 inhibitor that restores mitochondrial apoptosis priming, combined with high-dose cytarabine (± idarubicin/dexrazoxane or azacitidine) in a multicentre Phase 1 expansion in…
NCT03194932 - Trial result Spotlight
Acute myeloid leukemia and MDS
Ziftomenib (menin inhibitor) + venetoclax/azacitidine drives 46% CRc and frequent MRD negativity in R/R NPM1-mut AML
Ziftomenib is a potent, selective, oral menin inhibitor evaluated with venetoclax (BCL-2 inhibitor) plus azacitidine (hypomethylating agent) in KOMET-007, a Phase 1a/1b dose-escalation/expansion study in adults with…
NCT05735184 - New trial
Acute myeloid leukemia and MDS
Phase 1 CRD3874-SI allosteric STING agonist (TMEM173) in relapsed/refractory AML.
This Memorial Sloan Kettering Cancer Center–sponsored Phase 1 study tests escalating dose levels of CRD3874-SI, an intravenous small-molecule STING (TMEM173) agonist designed to activate innate immune signaling and…
NCT07661095 Exploratory - Trial result Spotlight
Acute myeloid leukemia and MDS
AZA/VEN + ivosidenib (IDH1 inhibitor) drives 95% ORR and 79% 3-year OS in unfit IDH1-mut AML
Azacitidine (DNA hypomethylating agent) + venetoclax (BCL-2 inhibitor) + ivosidenib (mutant IDH1 inhibitor) was evaluated as a frontline “triplet” in a multicenter Phase Ib/II study (NCT03471260) for newly diagnosed…
NCT03471260 - Trial result Spotlight
Acute myeloid leukemia and MDS
Mesutoclax (ICP-248), a selective BCL-2 inhibitor, drives 86% cCR with azacitidine in treatment-naïve AML
Mesutoclax (ICP-248) is a next-gen, highly selective BCL-2 inhibitor (positioned as a venetoclax-like backbone with a “cleaner” PK profile) tested with azacitidine in the ongoing global Phase 1 ICP-CL-01205…
NCT06656494 - Trial result
Acute myeloid leukemia and MDS
Quizartinib (FLT3 inhibitor) + decitabine/venetoclax drives 89% CR/CRi and 36.5-mo OS in older frontline FLT3-ITD AML
Quizartinib (QUIZ) is an oral, potent FLT3 inhibitor combined with decitabine (DNA hypomethylating agent) and venetoclax (BCL-2 inhibitor) in a Phase I/II study (NCT03661307) enrolling newly diagnosed FLT3-ITD AML…
NCT03661307 - Trial result
Acute myeloid leukemia and MDS
Revumenib (menin inhibitor; KMT2A/NPM1 program) maintenance post-alloSCT shows 89% 2-year OS with low 1-year relapse
Revumenib (Revuforj) is an oral menin inhibitor that disrupts the menin–KMT2A interaction and is FDA-approved (since November 15, 2024) for R/R acute leukemia with KMT2A rearrangement (≥1 year old); here it was used…
- Trial result
Acute myeloid leukemia and MDS
Luspatercept (TGF-β superfamily ligand trap) extends transfusion-independence vs epoetin alfa in ESA‑naive LR‑MDS; OS still trending better
Luspatercept (LUSPA), a recombinant fusion protein that binds select TGF-β superfamily ligands to promote late-stage erythroid maturation, was compared with epoetin alfa (EA; an ESA) in the Phase 3, open-label…
NCT03682536 - New trial Spotlight
Acute myeloid leukemia and MDS
Phase 1 FH-WT1-E50 WT1-targeted TCR‑T plus azacitidine for MRD+ AML
This Phase 1 study at Fred Hutchinson Cancer Center evaluates FH‑WT1‑E50, an autologous CD4+/CD8+ T-cell product genetically transduced to express an HLA‑A02:01–restricted, WT1-specific T-cell receptor (TCR‑T; MOA:…
NCT07645469 First-in-human - New trial
Acute myeloid leukemia and MDS
Phase 1b pivekimab sunirine CD123-targeted antibody–drug conjugate in pediatric R/R AML
AbbVie is running this open-label, single-arm Phase 1b study to characterize safety, tolerability, and pharmacokinetics of pivekimab sunirine in children with relapsed or refractory acute myeloid leukemia whose…
NCT07306832 Exploratory - New trial
Acute myeloid leukemia and MDS
Phase 2 venetoclax (BCL-2 inhibitor) vs gemtuzumab ozogamicin (CD33 ADC) with 7+3 in CBF-AML
This National Cancer Institute (NCI) MyeloMATCH Phase 2, randomized treatment substudy tests whether adding AbbVie/Genentech’s venetoclax (a small-molecule BCL-2 inhibitor that promotes apoptosis) to standard “7+3”…
NCT06917911 Exploratory - New trial
Acute myeloid leukemia and MDS
Phase 1 ATRN-119 ATR kinase inhibitor plus decitabine (DNA methyltransferase inhibitor) in TP53 AML/MDS.
This single-center, open-label Phase 1 study at Washington University School of Medicine (St. Louis) evaluates ATRN-119 (Aprea Therapeutics; an oral small-molecule inhibitor of ATR, a key DNA damage–response kinase)…
NCT07617363 Exploratory - New trial Spotlight
Acute myeloid leukemia and MDS
Phase 2 revumenib menin inhibitor triplet with azacitidine + venetoclax in KMT2A-rearranged AML.
UNC Lineberger’s RAVEN study (NCT07605949) evaluates an all-oral/SC intensive-lower regimen combining revumenib (a menin–KMT2A/MLL interaction inhibitor that downregulates leukemogenic HOX/MEIS programs) with…
NCT07605949 Exploratory - New trial
Acute myeloid leukemia and MDS
Phase 1/2 AdaptNK adaptive CMV-induced NK-cell immunotherapy (KIR–HLA mismatch bridge-to-transplant) in R/R AML.
This Phase 1/2, open-label, multi-institution study from Masonic Cancer Center (University of Minnesota) tests an allogeneic KIR–HLA–mismatched natural killer (NK) cell infusion, “AdaptNK,” followed by a short course…
NCT07591649 Exploratory - Trial result
Acute myeloid leukemia and MDS
Ivosidenib (mutant IDH1 inhibitor) + azacitidine drives a 74-month complete remission in IDH1-mutated elderly AML (case report)
Ivosidenib is an oral small-molecule inhibitor of mutant IDH1 used with the hypomethylating agent azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy (IC). In a…
NCT03173248 - New trial Spotlight
Acute myeloid leukemia and MDS
Phase 1 BSB-2002 mutant NPM1–targeted TCR-T therapy in relapsed/refractory AML
This Phase 1, open-label, dose-finding study evaluates BSB-2002, a personalized autologous T cell receptor–engineered T cell (TCR‑T) therapy whose introduced TCR recognizes a mutant NPM1 peptide presented by…
NCT07583303 First-in-human - New trial
Acute myeloid leukemia and MDS
Phase 1 ASTX727 (oral decitabine + cedazuridine; DNA hypomethylating agent + cytidine deaminase inhibitor) with filgrastim (G‑CSF)
This Phase 1, post–allogeneic hematopoietic cell transplant (HCT) maintenance study tests whether ASTX727 plus filgrastim can be given safely, and at what dose, to help prevent relapse in children with high-risk AML…
NCT07012044 Exploratory - New trial Spotlight
Acute myeloid leukemia and MDS
Phase 1 BSB-2002 mutant NPM1–targeted TCR-T cellular therapy in R/R AML
This Phase 1, multicenter, open-label, non-randomized dose-escalation/expansion study tests BSB-2002 for adults with relapsed or refractory acute myeloid leukemia whose leukemia harbors an NPM1 mutation and who are…
NCT07566585 First-in-human - New trial Spotlight
Acute myeloid leukemia and MDS
Phase 3 bleximenib (JNJ-75276617) menin–KMT2A interaction inhibitor added to intensive AML chemo
This randomized, double-blind, placebo-controlled Phase 3 trial tests whether adding bleximenib (JNJ-75276617)—an oral small-molecule inhibitor of the menin–KMT2A protein interaction—improves outcomes when layered…
NCT07223814 Registrational - New trial
Acute myeloid leukemia and MDS
Phase 1 ruxolitinib JAK1/2 inhibitor plus azacitidine DNA hypomethylating agent after alloHSCT
This Phase 1, single-site OHSU Knight Cancer Institute study tests safety, tolerability, and dose selection for post-transplant ruxolitinib (a JAK1/2 inhibitor; marketed as Jakafi by Incyte) given first as…
NCT07548983 Exploratory - New trial
Acute myeloid leukemia and MDS
Phase 1 PLM-102 FLT3 inhibitor (next-generation tyrosine kinase inhibitor) in R/R AML/MDS
This Phase 1, open-label, dose-escalation study at MD Anderson Cancer Center is designed to define the maximum tolerated dose and recommended Phase 2 dose of PLM-102, an oral small-molecule FLT3 tyrosine kinase…
NCT07549464 Exploratory - New trial
Acute myeloid leukemia and MDS
Phase 2 imetelstat telomerase inhibitor in MDS/MPN overlap syndromes and CMML.
This Phase 2, single-arm MD Anderson study tests imetelstat, a first-in-class telomerase inhibitor (an oligonucleotide that binds the telomerase RNA template to block telomere maintenance), given by IV infusion every…
NCT07549451 Exploratory
Looking for trials that fit a specific diagnosis? Read our guide on how to find a clinical trial, or subscribe to New in Oncology to get each issue by email.