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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)

RecruitingPhase 2

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

Who can join

Ages 18 to 59 · All sexes

Full eligibility criteria
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

About the study

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.

What is being tested

Sponsor: National Cancer Institute (NCI) · Participants: 162 · Started: Feb 2, 2027

Contact the study team

Official record on ClinicalTrials.gov — NCT06917911

Locations in the U.S.

AlabamaUniversity of Alabama at Birmingham Cancer Center, Birmingham
FloridaMemorial Hospital West, Pembroke Pines
IdahoKootenai Health - Coeur d'Alene, Coeur d'Alene
Kootenai Clinic Cancer Services - Post Falls, Post Falls
Kootenai Clinic Cancer Services - Sandpoint, Sandpoint
IllinoisNorthwestern University, Chicago
KansasUniversity of Kansas Clinical Research Center, Fairway
University of Kansas Cancer Center, Kansas City
University of Kansas Hospital-Indian Creek Campus, Overland Park
University of Kansas Hospital-Westwood Cancer Center, Westwood
KentuckyUniversity of Kentucky/Markey Cancer Center, Lexington
The James Graham Brown Cancer Center at University of Louisville, Louisville
UofL Health Medical Center Northeast, Louisville
MassachusettsTufts Medical Center, Boston
MichiganTrinity Health IHA Medical Group Hematology Oncology - Brighton, Brighton
Trinity Health IHA Medical Group Hematology Oncology - Canton, Canton
Trinity Health IHA Medical Group Hematology Oncology - Chelsea Hospital, Chelsea
Trinity Health Saint Mary Mercy Livonia Hospital, Livonia
Trinity Health Saint Joseph Mercy Oakland Hospital, Pontiac
Trinity Health IHA Medical Group Hematology Oncology Ann Arbor Campus, Ypsilanti
MississippiBaptist Memorial Hospital and Cancer Center-Golden Triangle, Columbus
Baptist Cancer Center-Grenada, Grenada
Baptist Memorial Hospital and Cancer Center-Union County, New Albany
Baptist Memorial Hospital and Cancer Center-Oxford, Oxford
Baptist Memorial Hospital and Cancer Center-Desoto, Southhaven
MissouriSiteman Cancer Center at Saint Peters Hospital, City of Saint Peters
Siteman Cancer Center at West County Hospital, Creve Coeur
Siteman Cancer Center at Christian Hospital, St Louis
Siteman Cancer Center-South County, St Louis
Washington University School of Medicine, St Louis
MontanaBillings Clinic Cancer Center, Billings
Bozeman Health Deaconess Hospital, Bozeman
Benefis Sletten Cancer Institute, Great Falls
Community Medical Center, Missoula
NebraskaNebraska Medicine-Bellevue, Bellevue
Nebraska Medicine-Village Pointe, Omaha
University of Nebraska Medical Center, Omaha
New MexicoUniversity of New Mexico Cancer Center, Albuquerque
New YorkRoswell Park Cancer Institute, Buffalo
University of Rochester, Rochester
State University of New York Upstate Medical University, Syracuse
Montefiore Medical Center - Moses Campus, The Bronx
North CarolinaDuke University Medical Center, Durham
OhioOhio State University Comprehensive Cancer Center, Columbus
OklahomaUniversity of Oklahoma Health Sciences Center, Oklahoma City
PennsylvaniaGeisinger Medical Center, Danville
Geisinger Wyoming Valley/Henry Cancer Center, Wilkes-Barre
South CarolinaPrisma Health Cancer Institute - Spartanburg, Boiling Springs
Prisma Health Cancer Institute - Easley, Easley
Prisma Health Cancer Institute - Butternut, Greenville
Prisma Health Cancer Institute - Eastside, Greenville
Prisma Health Cancer Institute - Faris, Greenville
Prisma Health Cancer Institute - Greer, Greer
Prisma Health Cancer Institute - Seneca, Seneca
TennesseeBaptist Memorial Hospital and Cancer Center-Collierville, Collierville
Baptist Memorial Hospital and Cancer Center-Memphis, Memphis
UtahHuntsman Cancer Institute/University of Utah, Salt Lake City
WashingtonSwedish Cancer Institute-Edmonds, Edmonds
Swedish Cancer Institute-Issaquah, Issaquah
Swedish Medical Center-First Hill, Seattle
WisconsinGundersen Lutheran Medical Center, La Crosse
Froedtert Menomonee Falls Hospital, Menomonee Falls
Medical College of Wisconsin, Milwaukee
Froedtert and MCW Moorland Reserve Health Center, New Berlin
Drexel Town Square Health Center, Oak Creek
Froedtert West Bend Hospital/Kraemer Cancer Center, West Bend

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.