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. 2023 May 29;15(11):2958. doi: 10.3390/cancers15112958

Table 1.

Selected phase 3 trials in AML.

NCT Number Study Name Study Design and Status Drugs Tested Drug Class Primary Outcome
NCT02013648 Randomized Phase III Study of Intensive Chemotherapy with or Without Dasatinib Randomized, open label
Active not recruiting		 Dasatinib Tyrosine Kinase inhibitor

  1. EFS
NCT04102020 Randomized, Double-Blind, 2-Arm, Multicenter, Phase 3 Study of Venetoclax and Oral Azacitidine Versus Oral Azacitidine as Maintenance Therapy for Patients with Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy (VIALE-M) Randomized, double blind
Active and recruiting		 Venetoclax
Azacitidine
CC-486		 BCL2 inhibitor
  • 1.

    DLT

  • 2.

    DLT of combination

  • 3.

    RFS
NCT04716114 A Phase 3, Open-Label, Multicenter, Randomized Study of SKLB1028 Versus Salvage Chemotherapy in Patients with FLT3-mutated Acute Myeloid Leukemia Refractory to or Relapsed After First-line Treatment (ALIVE) Randomized, open label
Active and recruiting		 SKLB1028 Multikinase inhibitor of EGFR, FLT3 and ABL
  • 1.

    CR/CRh rate

  • 2.

    OS
NCT05586074 HEC73543 Versus Salvage Chemotherapy in Relapsed or Refractory FLT3-ITD Acute Myeloid Leukemia: a Multicenter, Open-label, Randomized Phase 3 Trial Randomized, Open label
Active not yet recruiting		 Clifutinib FLT3 inhibitor

  1. OS

  2. CR/CRh rate
NCT04161885 A Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in Combination with Azacitidine After Allogeneic Stem Cell Transplantation in Subjects With Acute Myeloid Leukemia (AML) (VIALE-T) Randomized, Open label
Active, recruiting		 Venetoclax
Azacitidine		 BCL2 inhibitor

  1. DLT of combination

  2. RFS
NCT04628026 A Randomized, Placebo-Controlled Phase III Study of Induction and Consolidation Chemotherapy with Venetoclax in Adult Patients with Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with Excess Blasts-2 Randomized, double blind
Active, recruiting		 Venetoclax BCL2 inhibitor

  1. EFS

  2. DLT
NCT04571645 A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Dociparstat Sodium in Combination with Standard Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia Randomized, double blind
Active, not recruiting		 Dociparastat CXCR4/CXCL12 inhibitor

  1. OS
NCT02997202 A Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Trial of the FLT3 Inhibitor Gilteritinib Administered as Maintenance Therapy Following Allogeneic Transplant for Patients with FLT3/ITD AML Randomized, double blind
Active, not recruiting		 Gilteritinib FLT3 inhibitor

  1. RFS
NCT05429632 Randomized, Double-blind, Placebo-controlled, Multi-center Phase III Study to Evaluate the Efficacy and Safety of Mocravimod as Adjunctive and Maintenance Treatment in Adult AML Patients Undergoing Allogeneic HCT Randomized, double blind
Active, recruiting		 Macrovimod Sphingosine-1-phosphate receptor modulator

  1. RFS
NCT03258931 Phase III Randomized Study of Crenolanib Versus Midostaurin Administered Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed Subjects With FLT3 Mutated Acute Myeloid Leukemia Randomized, open label
Active, recruiting		 Crenolanib FLT3 inhibitor

  1. EFS
NCT04229979 A Randomized, Open-Label Study of the Efficacy and Safety of Galinpepimut-S (GPS) Maintenance Monotherapy Compared to Investigator’s Choice of Best Available Therapy in Subjects with Acute Myeloid Leukemia Who Have Achieved Complete Remission After Second-Line Salvage Therapy Randomized, Open label
Active, recruiting		 Galinpepimut-S WT-1 peptide vaccine

  1. OS
NCT05079230 A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Magrolimab Versus Placebo in Combination with Venetoclax and Azacitidine in Newly Diagnosed, Previously Untreated Patients with Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy Randomized, Double blind
Active, recruiting		 Magrolimab Anti-CD47

  1. CR

  2. OS
NCT03616470 A Phase III Randomized, Double-Blind Trial to Evaluate the Efficacy of Uproleselan Administered with Chemotherapy Versus Chemotherapy Alone in Patients With Relapsed/Refractory Acute Myeloid Leukemia Randomized, Double blind
Active, not recruiting		 Uproleselan E-selectin antagonist

  1. OS
NCT02668653 A Phase 3, Double-Blind, Placebo-controlled Study of Quizartinib Administered in Combination with Induction and Consolidation Chemotherapy, and Administered as Continuation Therapy in Subjects 18 to 75 Years Old with Newly Diagnosed FLT3-ITD (+) Acute Myeloid Leukemia (QuANTUM First) Randomized, Double blind
Active, not recruiting		 Quizartinib FLT3 inhibitor

  1. OS
NCT03250338 Phase III Randomized, Double-blind, Placebo-controlled Study Investigating the Efficacy of the Addition of Crenolanib to Salvage Chemotherapy Versus Salvage Chemotherapy Alone in Subjects ≤ 75 Years of Age with Relapsed/Refractory FLT3 Mutated Acute Myeloid Leukemia Randomized, double blind
Active, recruiting		 Crenolanib FLT3 inhibitor

  1. EFS
NCT04161885 A Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in Combination with Azacitidine After Allogeneic Stem Cell Transplantation in Subjects With Acute Myeloid Leukemia (AML) (VIALE-T) Randomized, open label
Active, recruiting		 Venetoclax
Azacitidine		 BCL2 inhibitor

  1. DLT

  2. RFS
NCT05020665 A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Entospletinib in Combination With Intensive Induction and Consolidation Chemotherapy in Adults With Newly Diagnosed Nucleophosmin 1-mutated Acute Myeloid Leukemia Randomized, double blind
Active, not recruiting		 Entospletinib spleen tyrosine kinase (SYK) inhibitor

  1. MRD negative CR
NCT04778397 A Phase 3, Randomized, Open-Label Study Evaluating the Safety and Efficacy of Magrolimab in Combination With Azacitidine Versus Physician’s Choice of Venetoclax in Combination With Azacitidine or Intensive Chemotherapy in Previously Untreated Patients With TP53 Mutant Acute Myeloid Leukemia ENHANCE-2 Randomized, Open label
Active, recruiting		 Magrolimab Anti-CD47

  1. OS
NCT02665065 A Multicenter, Pivotal Phase 3 Study of Iomab-B Prior to Allogeneic Hematopoietic Cell Transplant Versus Conventional Care in Older Subjects with Active, Relapsed or Refractory Acute Myeloid Leukemia (AML) (SIERRA) Randomized, Open label
Active, not recruiting		 Iomab-B Anti-CD45-131I apamistamab

  1. dCR
NCT03926624 Phase 3 Randomized Trial of DFP-10917 vs. Non-Intensive Reinduction (LoDAC, Azacitidine, Decitabine, Venetoclax Combination Regimens) or Intensive Reinduction (High & Intermediate Dose Cytarabine Regimens) for Acute Myelogenous Leukemia Patients in Second, Third, or Fourth Salvage Randomized, Open label
Active, recruiting		 DFP-10917 deoxycytidine nucleoside analogue

  1. CR

  2. Duration of CR
NCT03268954 A Phase 3, Randomized, Controlled, Open-label, Clinical Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Patients with Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Low-Blast Acute Myelogenous Leukemia Randomized, Open label
Active, not recruiting		 Pevonedistat NAE inhibitor

  1. EFS
NCT03092674 A Randomized Phase II/III Trial of “Novel Therapeutics” Versus Azacitidine in Newly Diagnosed Patients With Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS), Age 60 or Older LEAP: Less-Intense AML Platform Trial Randomized, Open label
Active, not recruiting		 Nivolumab PD-1 inhibitor

  1. OS
NCT03257241 A PALG Prospective Multicenter Clinical Trial to Compare the Efficacy of Two Standard Induction Therapies (DA-90 vs. DAC) and Two Standard Salvage Regimens (FLAG-IDA vs. CLAG-M) in AML Patients ≤ 60 Years Old Randomized, Open label
Active, recruiting		 DNR 90
DAC
CLAG-M
FLAG- IDA		 Anthracycline

  1. Induction regimen efficacy

  2. Salvage regimen efficacy

EFS—Event free survival, DLT—Dose limiting toxicity, RFS- Relapse free survival, CR Complete Remission, CRh—Complete remission hematological, OS—Overall Survival, MRD—minimal residual disease, dCR—Durable Complete Remission.