Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MYELOMATCH Treatment Trial)

September 1, 2026 updated by: National Cancer Institute (NCI)

Phase II Study of Cytarabine + Daunorubicin (7 + 3) + Gemtuzumab Ozogamicin vs. Cytarabine + Daunorubicin (7 + 3) + Venetoclax for the Treatment of Newly Diagnosed Core Binding Factor Acute Myeloid Leukemia (CBF-AML) in Younger Adults: A MyeloMATCH Substudy

This phase II MYELOMATCH treatment trial compares the effect of venetoclax to gemtuzumab ozogamicin, when given with cytarabine and daunorubicin ("7+3" regimen), for the treatment of patients with core binding factor acute myeloid leukemia (CBF-AML). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Gemtuzumab ozogamicin is a monoclonal antibody, called gemtuzumab, linked to an antitumor antibiotic drug, called ozogamicin. Gemtuzumab is a form of targeted therapy because it attaches to specific molecules (receptors) on the surface of cancer cells, known as CD33 receptors, and delivers ozogamicin to kill them. Chemotherapy drugs, such as cytarabine and daunorubicin work in different ways to stop the growth of cancer cells either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving venetoclax with cytarabine and daunorubicin may have fewer side effects and be as effective or better than the combination with gemtuzumab ozogamicin in treating patients with core binding factor AML.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

PRIMARY OBJECTIVE:

I. Compare the rates of complete remission (CR) without measurable residual disease (CRMRD-) by multiparameter flow cytometry following induction therapy between the two treatment arms in each cohort separately.

SECONDARY OBJECTIVES:

I. To compare the rates of CR and composite complete remission (CRc) (CR+complete remission with incomplete hematologic recovery [CRi]+complete remission with partial hematologic recovery [CRh]) between the treatment arms.

II. To compare the overall survival (OS) between the treatment arms. III. To compare the event-free survival (EFS). IV. To compare the cumulative incidence of relapse (CIR). V. To compare the cumulative incidence of death (CID) between the treatment arms.

VI. To compare the rate of early death at 30 days and 60 days between the treatment arms.

VII. To assess the rate and frequency of adverse events between treatment arms. VIII. To evaluate mutant RAS and mutant KIT as predictive biomarkers for CRMRD- rate in CBF AML.

EXPLORATORY OBJECTIVE:

I. To compare MRD (and its clinical implication, e.g., relapse rates) between flow cytometry (FC) and next generation sequencing (NGS)-based (RUNX1::RUNX1T1 or CBFB::MYH11).

CORRELATIVE OBJECTIVES:

I. To evaluate the frequency and clinical impact of variant allele frequency (VAF) of KIT mutation, KIT mutations in different exons (e.g., exon 8 or 17), CD33 expression, additional (secondary) mutations and cytogenetic abnormalities.

II. To evaluate the differences in clinical and molecular outcomes in patients with RUNX1::RUNX1T1 mutated versus CBFB::MYH11 mutated CBF AML.

OUTLINE: Patients are randomized to 1 of 2 regimens.

REGIMEN 1: Patients receive gemtuzumab ozogamicin intravenously (IV) on day 1, 2 or 3 and on day 4, 5, or 6 (NOTE: two doses must be at least 3 days apart), as well as cytarabine IV, continuously, on days 1-7 and daunorubicin IV on days 1-3 in the absence of disease progression or unacceptable toxicity. Patients then undergo standard of care consolidation/post-remission treatment at the discretion of the treating physician. Patients undergo echocardiography or multigated acquisition (MUGA) scan during screening, as well as bone marrow aspiration and blood sample collection throughout the study.

REGIMEN 2: Patients receive venetoclax orally (PO) once daily (QD) on days 1-11, cytarabine IV, continuously, on days 2-8 and daunorubicin IV on days 2-4 in the absence of disease progression or unacceptable toxicity. Patients then undergo standard of care consolidation/post-remission treatment at the discretion of the treating physician. Patients undergo echocardiography or MUGA scan during screening and bone marrow aspiration, as well as blood sample collection throughout the study.

After completion of study treatment, patients are followed up at relapse and every 3 months for 2 years, then every 6 months until 5 years.

Study Type

Interventional

Enrollment (Estimated)

162

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Pembroke Pines, Florida, United States, 33028
        • Recruiting
        • Memorial Hospital West
        • Contact:
          • Site Public Contact
          • Phone Number: 954-265-4325
        • Principal Investigator:
          • Yehuda E. Deutsch
    • Idaho
      • Coeur d'Alene, Idaho, United States, 83814
        • Recruiting
        • Kootenai Health - Coeur d'Alene
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Post Falls, Idaho, United States, 83854
        • Recruiting
        • Kootenai Clinic Cancer Services - Post Falls
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Sandpoint, Idaho, United States, 83864
        • Recruiting
        • Kootenai Clinic Cancer Services - Sandpoint
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Northwestern University
        • Contact:
        • Principal Investigator:
          • Yuchen Liu
    • Kansas
      • Fairway, Kansas, United States, 66205
        • Recruiting
        • University of Kansas Clinical Research Center
        • Contact:
        • Principal Investigator:
          • Jesus Gonzalez Lugo
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Cancer Center
        • Contact:
        • Principal Investigator:
          • Jesus Gonzalez Lugo
      • Overland Park, Kansas, United States, 66211
        • Recruiting
        • University of Kansas Hospital-Indian Creek Campus
        • Contact:
        • Principal Investigator:
          • Jesus Gonzalez Lugo
      • Westwood, Kansas, United States, 66205
        • Recruiting
        • University of Kansas Hospital-Westwood Cancer Center
        • Contact:
        • Principal Investigator:
          • Jesus Gonzalez Lugo
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • University of Kentucky/Markey Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 859-257-3379
        • Principal Investigator:
          • Fevzi Yalniz
      • Louisville, Kentucky, United States, 40202
        • Recruiting
        • The James Graham Brown Cancer Center at University of Louisville
        • Principal Investigator:
          • Mohamed M. Hegazi
        • Contact:
          • Site Public Contact
          • Phone Number: 502-562-3429
      • Louisville, Kentucky, United States, 40245
        • Recruiting
        • UofL Health Medical Center Northeast
        • Principal Investigator:
          • Mohamed M. Hegazi
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02111
    • Michigan
      • Brighton, Michigan, United States, 48114
        • Recruiting
        • Trinity Health IHA Medical Group Hematology Oncology - Brighton
        • Contact:
        • Principal Investigator:
          • Tareq Al baghdadi
      • Canton, Michigan, United States, 48188
        • Recruiting
        • Trinity Health IHA Medical Group Hematology Oncology - Canton
        • Contact:
        • Principal Investigator:
          • Tareq Al baghdadi
      • Chelsea, Michigan, United States, 48118
        • Recruiting
        • Trinity Health IHA Medical Group Hematology Oncology - Chelsea Hospital
        • Contact:
        • Principal Investigator:
          • Tareq Al baghdadi
      • Livonia, Michigan, United States, 48154
        • Recruiting
        • Trinity Health Saint Mary Mercy Livonia Hospital
        • Contact:
        • Principal Investigator:
          • Tareq Al baghdadi
      • Pontiac, Michigan, United States, 48341
        • Recruiting
        • Trinity Health Saint Joseph Mercy Oakland Hospital
        • Contact:
        • Principal Investigator:
          • Tareq Al baghdadi
      • Ypsilanti, Michigan, United States, 48197
        • Recruiting
        • Trinity Health IHA Medical Group Hematology Oncology Ann Arbor Campus
        • Contact:
        • Principal Investigator:
          • Tareq Al baghdadi
    • Mississippi
      • Columbus, Mississippi, United States, 39705
        • Recruiting
        • Baptist Memorial Hospital and Cancer Center-Golden Triangle
        • Principal Investigator:
          • Salil Goorha
        • Contact:
      • Grenada, Mississippi, United States, 38901
        • Recruiting
        • Baptist Cancer Center-Grenada
        • Principal Investigator:
          • Salil Goorha
        • Contact:
      • New Albany, Mississippi, United States, 38652
        • Recruiting
        • Baptist Memorial Hospital and Cancer Center-Union County
        • Principal Investigator:
          • Salil Goorha
        • Contact:
      • Oxford, Mississippi, United States, 38655
        • Recruiting
        • Baptist Memorial Hospital and Cancer Center-Oxford
        • Principal Investigator:
          • Salil Goorha
        • Contact:
      • Southhaven, Mississippi, United States, 38671
        • Recruiting
        • Baptist Memorial Hospital and Cancer Center-Desoto
        • Principal Investigator:
          • Salil Goorha
        • Contact:
    • Missouri
      • City of Saint Peters, Missouri, United States, 63376
        • Recruiting
        • Siteman Cancer Center at Saint Peters Hospital
        • Contact:
        • Principal Investigator:
          • Ramzi Abboud
      • Creve Coeur, Missouri, United States, 63141
        • Recruiting
        • Siteman Cancer Center at West County Hospital
        • Contact:
        • Principal Investigator:
          • Ramzi Abboud
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine
        • Contact:
        • Principal Investigator:
          • Ramzi Abboud
      • St Louis, Missouri, United States, 63129
        • Recruiting
        • Siteman Cancer Center-South County
        • Contact:
        • Principal Investigator:
          • Ramzi Abboud
      • St Louis, Missouri, United States, 63136
        • Recruiting
        • Siteman Cancer Center at Christian Hospital
        • Contact:
        • Principal Investigator:
          • Ramzi Abboud
    • Montana
      • Billings, Montana, United States, 59101
        • Recruiting
        • Billings Clinic Cancer Center
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Bozeman, Montana, United States, 59715
        • Recruiting
        • Bozeman Health Deaconess Hospital
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Great Falls, Montana, United States, 59405
        • Recruiting
        • Benefis Sletten Cancer Institute
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Missoula, Montana, United States, 59804
        • Recruiting
        • Community Medical Center
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
    • Nebraska
      • Bellevue, Nebraska, United States, 68123
        • Recruiting
        • Nebraska Medicine-Bellevue
        • Contact:
        • Principal Investigator:
          • Michael Haddadin
      • Omaha, Nebraska, United States, 68198
        • Recruiting
        • University of Nebraska Medical Center
        • Contact:
        • Principal Investigator:
          • Michael Haddadin
      • Omaha, Nebraska, United States, 68118
        • Recruiting
        • Nebraska Medicine-Village Pointe
        • Contact:
          • Site Public Contact
          • Phone Number: 402-559-5600
        • Principal Investigator:
          • Michael Haddadin
    • New York
      • Buffalo, New York, United States, 14263
        • Recruiting
        • Roswell Park Cancer Institute
        • Principal Investigator:
          • Eunice S. Wang
        • Contact:
      • Rochester, New York, United States, 14642
        • Recruiting
        • University of Rochester
        • Contact:
          • Site Public Contact
          • Phone Number: 585-275-5830
        • Principal Investigator:
          • Bassil Botros
      • Syracuse, New York, United States, 13210
        • Recruiting
        • State University of New York Upstate Medical University
        • Principal Investigator:
          • Teresa C. Gentile
        • Contact:
          • Site Public Contact
          • Phone Number: 315-464-5476
      • The Bronx, New York, United States, 10467
        • Recruiting
        • Montefiore Medical Center - Moses Campus
        • Principal Investigator:
          • Ioannis Mantzaris
        • Contact:
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Recruiting
        • University of Oklahoma Health Sciences Center
        • Contact:
        • Principal Investigator:
          • Zimu Gong
    • Pennsylvania
      • Wilkes-Barre, Pennsylvania, United States, 18711
        • Recruiting
        • Geisinger Wyoming Valley/Henry Cancer Center
        • Contact:
        • Principal Investigator:
          • Joseph J. Vadakara
    • South Carolina
      • Boiling Springs, South Carolina, United States, 29316
        • Recruiting
        • Prisma Health Cancer Institute - Spartanburg
        • Principal Investigator:
          • Suzanne R. Fanning
        • Contact:
      • Easley, South Carolina, United States, 29640
        • Recruiting
        • Prisma Health Cancer Institute - Easley
        • Principal Investigator:
          • Suzanne R. Fanning
        • Contact:
      • Greenville, South Carolina, United States, 29605
        • Recruiting
        • Prisma Health Cancer Institute - Faris
        • Principal Investigator:
          • Suzanne R. Fanning
        • Contact:
      • Greenville, South Carolina, United States, 29605
        • Recruiting
        • Prisma Health Cancer Institute - Butternut
        • Principal Investigator:
          • Suzanne R. Fanning
        • Contact:
      • Greenville, South Carolina, United States, 29615
        • Recruiting
        • Prisma Health Cancer Institute - Eastside
        • Principal Investigator:
          • Suzanne R. Fanning
        • Contact:
      • Greer, South Carolina, United States, 29650
        • Recruiting
        • Prisma Health Cancer Institute - Greer
        • Principal Investigator:
          • Suzanne R. Fanning
        • Contact:
      • Seneca, South Carolina, United States, 29672
        • Recruiting
        • Prisma Health Cancer Institute - Seneca
        • Principal Investigator:
          • Suzanne R. Fanning
        • Contact:
    • Tennessee
      • Collierville, Tennessee, United States, 38017
        • Recruiting
        • Baptist Memorial Hospital and Cancer Center-Collierville
        • Principal Investigator:
          • Salil Goorha
        • Contact:
      • Memphis, Tennessee, United States, 38120
        • Recruiting
        • Baptist Memorial Hospital and Cancer Center-Memphis
        • Principal Investigator:
          • Salil Goorha
        • Contact:
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Recruiting
        • Huntsman Cancer Institute/University of Utah
        • Contact:
        • Principal Investigator:
          • Paul J. Shami
    • Wisconsin
      • La Crosse, Wisconsin, United States, 54601
        • Recruiting
        • Gundersen Lutheran Medical Center
        • Contact:
        • Principal Investigator:
          • Michael O. Ojelabi

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • GENERAL MYELOMATCH CRITERIA: Patients must be registered to the Master Screening and Reassessment Protocol, MYELOMATCH, and assigned to this protocol by the MATCHBox Treatment Verification Team

  • GENERAL MYELOMATCH CRITERIA: Participants must not have received prior anti-cancer therapy for AML or myelodysplastic syndrome (MDS)

    • Note: Hydroxyurea to control the white blood cell count (WBC) and cytarabine up to 1g for urgent cytoreduction is allowed.
    • Note: Prior erythroid stimulating agent (ESA) is not considered prior therapy for the purposes of eligibility
  • GENERAL MYELOMATCH CRITERIA: Participants must not receive any cytarabine-containing therapy other than up to 1g of cytarabine, which is allowed for urgent cytoreduction. Hydroxyurea, all-trans retinoic acid (ATRA), BCR-ABL directed tyrosine kinase inhibitor, erythropoiesis-stimulating agent, thrombopoietin receptor agonist and lenalidomide is allowed
  • Diagnosis of AML with t(8;21)(q22;q22.1)/RUNX1::RUNX1T1 or AML with inv(16)(p13.1q22) or t(16;16)(p13.1;q22)/CBFB::MYH11. No FLT3 mutation (these patients should be considered for a FLT3-focused MYELOMATCH study)
  • Secondary CBF-AML (e.g., prior pre-leukemic hematologic malignancy or history of chemotherapy/radiation therapy) is allowed.
  • No prior AML or MDS-directed therapy except for urgent treatment of leukocytosis with leukapheresis, cytarabine, and hydroxyurea, Prior intrathecal chemotherapy for central nervous system (CNS) involvement of AML is permitted
  • Age 18-59 years
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 3
  • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) (unless patient has a history of Gilbert syndrome and direct bilirubin is ≤ 1.5 x ULN)
  • Aspartate aminotransferase (AST)(serum glutamic oxaloacetic transaminase [SGOT])/ alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase [SGPT]) ≤ 3 x upper limit of normal (ULN)
  • Glomerular filtration rate (GFR) ≥ 30 mL/min/1.73m^2
  • Not pregnant and not nursing, because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown. Therefore, for women of childbearing potential only, a negative urine or serum pregnancy test done ≤ 7 days prior to registration is required
  • Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
  • Participants with CNS disease are eligible for this trial and will be treated according to institutional guidelines with intrathecal chemotherapy for this aspect of their disease
  • Patients with known HIV infection on effective anti-retroviral therapy with undetectable viral load within 6 months prior to registration are eligible for this trial
  • For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
  • Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
  • Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class II or better
  • No known medical condition causing an inability to swallow oral formulations of agents

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Regimen 1 (gemtuzumab ozogamicin 7+3)
Patients receive gemtuzumab ozogamicin IV on day 1, 2 or 3 and on day 4, 5, or 6 (NOTE: two doses must be at least 3 days apart), as well as cytarabine IV, continuously, on days 1-7 and daunorubicin IV on days 1-3 in the absence of disease progression or unacceptable toxicity. Patients then undergo standard of care consolidation/post-remission treatment at the discretion of the treating physician. Patients undergo echocardiography or MUGA scan during screening, as well as bone marrow aspiration and blood sample collection throughout the study.
Drug: Cytarabine
Given IV
Other Names:
  • .beta.-Cytosine arabinoside
  • 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone
  • 1-.beta.-D-Arabinofuranosylcytosine
  • 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone
  • 1-Beta-D-arabinofuranosylcytosine
  • 1.beta.-D-Arabinofuranosylcytosine
  • 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl-
  • 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl-
  • Alexan
  • Ara-C
  • ARA-cell
  • Arabine
  • Arabinofuranosylcytosine
  • Arabinosylcytosine
  • Aracytidine
  • Aracytin
  • Aracytine
  • Beta-Cytosine Arabinoside
  • CHX-3311
  • Cytarabinum
  • Cytarbel
  • Cytosar
  • Cytosine Arabinoside
  • Cytosine-.beta.-arabinoside
  • Cytosine-beta-arabinoside
  • Erpalfa
  • Starasid
  • Tarabine PFS
  • U 19920
  • U-19920
  • Udicil
  • WR-28453
Drug: Daunorubicin Hydrochloride
Given IV
Other Names:
  • Cerubidin
  • Cerubidine
  • Cloridrato de Daunorubicina
  • Daunoblastin
  • Daunoblastina
  • Daunoblastine
  • Daunomycin Hydrochloride
  • Daunomycin, hydrochloride
  • Daunorubicin.HCl
  • Daunorubicini Hydrochloridum
  • FI-6339
  • Ondena
  • RP-13057
  • Rubidomycin Hydrochloride
  • Rubilem
Procedure: Biospecimen Collection
Undergo blood sample collection
Other Names:
  • Biological Sample Collection
  • Biospecimen Collected
  • Specimen Collection
  • Sample Collection
Drug: Gemtuzumab Ozogamicin
Given IV
Other Names:
  • Mylotarg
  • Calicheamicin-Conjugated Humanized Anti-CD33 Monoclonal Antibody
  • CDP-771
  • CMA-676
  • gemtuzumab
  • hP67.6-Calicheamicin
  • WAY-CMA-676
Procedure: Bone Marrow Aspiration
Undergo bone marrow aspiration
Procedure: Multigated Acquisition Scan
Undergo MUGA scan
Other Names:
  • Blood Pool Scan
  • Equilibrium Radionuclide Angiography
  • Gated Blood Pool Imaging
  • MUGA
  • Radionuclide Ventriculography
  • RNVG
  • SYMA Scanning
  • Synchronized Multigated Acquisition Scanning
  • MUGA Scan
  • Multi-Gated Acquisition Scan
  • Radionuclide Ventriculogram Scan
  • Gated Heart Pool Scan
  • RNV Scan
Procedure: Echocardiography Test
Undergo echocardiography
Other Names:
  • Echocardiography
  • EC
Experimental: Regimen 2 (Venetoclax, 7+3)
Patients receive venetoclax orally (PO) once daily (QD) on days 1-11, cytarabine IV, continuously, on days 2-8 and daunorubicin IV on days 2-4 in the absence of disease progression or unacceptable toxicity. Patients then undergo standard of care consolidation/post-remission treatment at the discretion of the treating physician. Patients undergo echocardiography or MUGA scan during screening and bone marrow aspiration, as well as blood sample collection throughout the study.
Drug: Cytarabine
Given IV
Other Names:
  • .beta.-Cytosine arabinoside
  • 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone
  • 1-.beta.-D-Arabinofuranosylcytosine
  • 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone
  • 1-Beta-D-arabinofuranosylcytosine
  • 1.beta.-D-Arabinofuranosylcytosine
  • 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl-
  • 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl-
  • Alexan
  • Ara-C
  • ARA-cell
  • Arabine
  • Arabinofuranosylcytosine
  • Arabinosylcytosine
  • Aracytidine
  • Aracytin
  • Aracytine
  • Beta-Cytosine Arabinoside
  • CHX-3311
  • Cytarabinum
  • Cytarbel
  • Cytosar
  • Cytosine Arabinoside
  • Cytosine-.beta.-arabinoside
  • Cytosine-beta-arabinoside
  • Erpalfa
  • Starasid
  • Tarabine PFS
  • U 19920
  • U-19920
  • Udicil
  • WR-28453
Drug: Daunorubicin Hydrochloride
Given IV
Other Names:
  • Cerubidin
  • Cerubidine
  • Cloridrato de Daunorubicina
  • Daunoblastin
  • Daunoblastina
  • Daunoblastine
  • Daunomycin Hydrochloride
  • Daunomycin, hydrochloride
  • Daunorubicin.HCl
  • Daunorubicini Hydrochloridum
  • FI-6339
  • Ondena
  • RP-13057
  • Rubidomycin Hydrochloride
  • Rubilem
Procedure: Biospecimen Collection
Undergo blood sample collection
Other Names:
  • Biological Sample Collection
  • Biospecimen Collected
  • Specimen Collection
  • Sample Collection
Drug: Venetoclax
Given PO
Other Names:
  • Venclexta
  • ABT-0199
  • ABT-199
  • ABT199
  • GDC-0199
  • RG7601
  • Venclyxto
  • ABT 199
  • GDC 0199
  • GDC0199
Procedure: Bone Marrow Aspiration
Undergo bone marrow aspiration
Procedure: Multigated Acquisition Scan
Undergo MUGA scan
Other Names:
  • Blood Pool Scan
  • Equilibrium Radionuclide Angiography
  • Gated Blood Pool Imaging
  • MUGA
  • Radionuclide Ventriculography
  • RNVG
  • SYMA Scanning
  • Synchronized Multigated Acquisition Scanning
  • MUGA Scan
  • Multi-Gated Acquisition Scan
  • Radionuclide Ventriculogram Scan
  • Gated Heart Pool Scan
  • RNV Scan
Procedure: Echocardiography Test
Undergo echocardiography
Other Names:
  • Echocardiography
  • EC

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete remission without measurable residual disease (CRMRD-)
Time Frame: At end of induction (Up to 28 days)
MRD will be evaluated by Molecular Diagnostics Network flow cytometry and will be defined at a threshold of ≤ 10^-3.
At end of induction (Up to 28 days)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Rate of complete remission (CR)
Time Frame: At the end of treatment (Up to 28 days)
Defined as the number of evaluable patients achieving a response of CR per European Leukemia Network (ELN) 2022 criteria at the end of treatment divided by the total number of evaluable patients. Rates of response will be compared between treatment arms using a chi-square test (or Fisher's exact test as needed). Point estimates will be generated for CR rate within each arm along with 95% confidence intervals using the Clopper-Pearson method.
At the end of treatment (Up to 28 days)
Rate of composite CR
Time Frame: At the end of treatment (Up to 28 days)
Defined as the number of evaluable patients achieving a response of CR or complete remission with incomplete hematologic recovery (CRi) or complete remission with partial hematologic recovery (CRh) per ELN 2022 criteria at the end of treatment divided by the total number of evaluable patients. Rates of response will be compared between treatment arms using a chi-square test (or Fisher's exact test as needed). Point estimates will be generated for CR rate within each arm along with 95% confidence intervals using the Clopper-Pearson method.
At the end of treatment (Up to 28 days)
Overall survival (OS)
Time Frame: From registration until death due to any cause, up to 5 years
The distribution of OS will be estimated using method of Kaplan-Meier. The median OS and 95% confidence interval will be reported.
From registration until death due to any cause, up to 5 years
Event free survival (EVS)
Time Frame: From randomization to induction failure, hematologic relapse from CR/CRh/CRi or death from any cause, whichever occurs first, up to 5 years
Induction failure is defined as not achieving either CR, CRh or CRi by after induction therapy. The distribution of EFS will be estimated using method of Kaplan-Meier, and compared using log-rank tests. The median EFS and the corresponding 95% confidence interval will be reported.
From randomization to induction failure, hematologic relapse from CR/CRh/CRi or death from any cause, whichever occurs first, up to 5 years
Cumulative incidence of relapse (CIR)
Time Frame: From the date of achievement of a remission until the date of hematologic relapse, up to 5 years
The distribution of CIR will be estimated using Fine-Gray model and reported via hazard ratios and 95% confidence interval with p values.
From the date of achievement of a remission until the date of hematologic relapse, up to 5 years
Cumulative incidence of death (CID)
Time Frame: From the date of achievement of a remission to death without prior relapse, up to 5 years
The distribution of CID will be estimated using Fine-Gray model and reported via hazard ratios and 95% confidence interval with p values.
From the date of achievement of a remission to death without prior relapse, up to 5 years
Rate of early death at 30 days (ED-30)
Time Frame: At 30 days
ED-30 is defined as the total number of patients who died on or before day 30 from randomization divided by the total number of evaluable patients. Rate of early death at day 30 will be compared between treatment arms using a chi-square test (or Fisher's exact test as needed). Point estimates will be generated for ED-30 rate within each arm along with 95% confidence intervals using the Clopper-Pearson method.
At 30 days
Rate of early death at 60 days (ED-60)
Time Frame: At 60 days
ED-60 is defined as the total number of patients who died on or before day 60 from randomization divided by the total number of evaluable patients. Rate of early death at day 60 will be compared between treatment arms using a chi-square test (or Fisher's exact test as needed). Point estimates will be generated for ED-60 rate within each arm along with 95% confidence intervals using the Clopper-Pearson method.
At 60 days
Incidence of adverse events
Time Frame: Up to 5 years
As per national Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns. The incidence of severe (grade 3+) adverse events or toxicities will be described for each treatment arm and will also be compared between the arms. Fisher's exact tests will be used to quantitatively compare the incidence of severe as well as specific toxicities of interest between the treatment arms and we will graphically assess differences in maximum grades observed for toxicities between the arms.
Up to 5 years
Evaluate mutant RAS and mutant KIT as predictive biomarkers for CRMRD- rate
Time Frame: Up to 5 years
In cohort 1, subgroup analysis will be conducted to evaluate the treatment effect among RAS mutated patients using Kaplan Meier methods and log-rank tests; similar subgroup analysis will be done among KIT mutated patients.
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Cancer Institute (NCI)

Investigators

  • Principal Investigator: Celalettin Ustun, Alliance for Clinical Trials in Oncology

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

February 2, 2027

Primary Completion (Estimated)

November 25, 2027

Study Completion (Estimated)

November 25, 2027

Study Registration Dates

First Submitted

April 8, 2025

First Submitted That Met QC Criteria

April 8, 2025

First Posted (Actual)

April 9, 2025

Study Record Updates

Last Update Posted (Actual)

September 2, 2026

Last Update Submitted That Met QC Criteria

September 1, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NCI-2025-02426 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
  • U10CA180821 (U.S. NIH Grant/Contract)
  • MM1YA-A04 (Other Identifier: CTEP)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe