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NCT05600894Active, not recruitingPhase 2

Venetoclax in Combination With ASTX727 for the Treatment of Chronic Myelomonocytic Leukemia and Other Myelodysplastic Syndrome/Myeloproliferative Neoplasm

Venetoclax In Combination With ASTX727, an All-ORal TherapY for Chronic Myelomonocytic Leukemia and Other MDS/MPN With Excess Blasts (VICTORY-MDS/MPN): a Randomized, Phase 2 Trial

Source: ClinicalTrials.gov · Last updated 19 Aug 2026

Brief summary (registry, in English)

This phase II trial tests whether decitabine and cedazuridine (ASTX727) in combination with venetoclax work better than ASTX727 alone at decreasing symptoms of bone marrow cancer in patients with chronic myelomonocytic leukemia (CMML), myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) with excess blasts. Blasts are immature blood cells. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. 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. The combination of ASTX727 and venetoclax may be more effective in reducing the cancer signs and symptoms in patients with CMML, or MDS/MPN with excess blasts.

Status (registry)
Active, not recruiting
Phase (registry)
Phase 2
Study type
Interventional
Enrollment
132 (estimated)
First submitted
27 Oct 2022
Start date
27 Jun 2023
Primary completion date
31 Aug 2027
Lead sponsor
National Cancer Institute (NCI)
NCT number
NCT05600894

What you commit to

  • Study type: Interventional · Phase 2
  • Planned participants: 132
  • From 27 Jun 2023 to the primary completion date, 31 Aug 2027
  • 34 sites in 2 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • More than one cycle of previous MDS/MPN-directed therapy, or MDS-directed therapy including lenalidomide and hypomethylating agent (HMAs) such as decitabine or azacitidine, excluding hydroxyurea. Prior use of erythropoietin stimulating agents (ESA) and thrombopoietic agents is allowed, but must be discontinued 4 weeks prior to study treatment
  • Patients currently or previously receiving an investigational agent or device within 4 weeks of the first dose of treatment
  • Patients with symptomatic uncontrolled central nervous system (CNS) disease. Imaging to confirm the absence of brain metastases is not required. Patients with spinal cord compression unless considered to have received definitive treatment for this and evidence of clinically stable disease for 28 days
  • Patients who have consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or starfruit within 3 days prior to the initiation of study treatment and are unwilling to discontinue consumption of these throughout the receipt of study drug
  • Patients with a prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or assessment of the investigational regimen

Other illness

  • Patients with need for emergent disease-directed therapy excluding hydroxyurea
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to ASTX727 or venetoclax
  • Patients with uncontrolled intercurrent illness (e.g. requiring intravenous therapy) at the discretion of the investigator
  • Patients with any other medical condition for which the expected survival is below 12 months
  • Patients with uncontrolled infection at the time of study entry

Lab values

  • Pregnant women are excluded from this study because venetoclax and ASTX727 have the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with venetoclax, breastfeeding should be discontinued if the mother is treated with venetoclax. These potential risks may also apply to other agents used in this study. Patients must be post-menopausal or with evidence of non-childbearing status for women of childbearing potential: negative urine or serum pregnancy test within 28 days of study treatment and confirmed prior to treatment on day 1
    • Post-menopausal is defined as:
    • Amenorrheic for 1 year or more following cessation of exogenous hormonal treatments
    • Luteinizing hormone (LH) and follicle stimulating hormone (FSH) levels in the post-menopausal range for women under 50 years of age
    • Radiation-induced oophorectomy with last menses \> 1 year ago
    • Chemotherapy-induced menopause with \> 1 year interval since last menses
    • Surgical sterilization (bilateral oophorectomy or hysterectomy)
    • Women of child-bearing potential must agree to use adequate contraception (hormonal birth control or abstinence) prior to study entry and for the duration of study participation, and for 6 months following completion of study treatment. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception (latex or synthetic condom or abstinence) prior to the study, for the duration of study participation, and 3 months after completion of venetoclax and ASTX727 administration

Who the study is looking for

  • A diagnosis of an MDS/MPN "overlap" syndrome with \>= 5% marrow blasts (including monocytic blast equivalent in case of CMML). Hydroxyurea may be used to control counts up until the start of therapy
  • White blood cell (WBC) \< 25,000/mm\^3. Treatment with hydroxyurea is permitted to lower the WBC to reach this criterion
  • Age \>= 18 years. Because no dosing or adverse event data are currently available on the use of ASTX727 in combination with venetoclax in patients \< 18 years of age, children are excluded from this study
  • Eastern Cooperative Oncology Group (ECOG) performance status =\< 2
  • Total bilirubin =\< 1.5 x upper limit of normal (ULN) (unless considered due to Gilbert's syndrome)
  • Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 3.0 x institutional ULN OR =\< 5.0 x institutional ULN for patients with liver metastases
  • Glomerular filtration rate (GFR) \>= 30 mL/min/1.73 m\^2
  • Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months 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 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. Hormonal therapy for prior or concurrent malignancy is allowed
  • 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 2B or better
  • Ability to understand and the willingness to sign a written informed consent document. Participants with impaired decision-making capacity (IDMC) who have a legally authorized representative (LAR) and/or family member available will also be eligible
  • Ability to swallow pills

Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (34)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
University Health Network, Princess Margaret Hospital
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TorontoCanadaUnknown status
University of Maryland/Greenebaum Cancer Center
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BaltimoreUnited StatesUnknown status
Johns Hopkins University/Sidney Kimmel Cancer Center
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BaltimoreUnited StatesUnknown status
Beth Israel Deaconess Medical Center
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BostonUnited StatesUnknown status
UNC Lineberger Comprehensive Cancer Center
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Chapel HillUnited StatesUnknown status
University of Virginia Cancer Center
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CharlottesvilleUnited StatesUnknown status
University of Chicago Comprehensive Cancer Center
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ChicagoUnited StatesUnknown status
Northwestern University
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ChicagoUnited StatesUnknown status
University of Cincinnati Cancer Center-UC Medical Center
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CincinnatiUnited StatesUnknown status
Ohio State University Comprehensive Cancer Center
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ColumbusUnited StatesUnknown status
UC Irvine Health Cancer Center-Newport
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Costa MesaUnited StatesUnknown status
UCI Health - Chao Family Comprehensive Cancer Center and Ambulatory Care
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IrvineUnited StatesUnknown status
Mayo Clinic in Florida
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JacksonvilleUnited StatesUnknown status
University of Kansas Cancer Center
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Kansas CityUnited StatesUnknown status
UCI Health - Laguna Hills
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Laguna HillsUnited StatesUnknown status
Los Angeles General Medical Center
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Los AngelesUnited StatesUnknown status
USC / Norris Comprehensive Cancer Center
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Los AngelesUnited StatesUnknown status
Yale University
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New HavenUnited StatesUnknown status
UC Comprehensive Cancer Center at Silver Cross
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New LenoxUnited StatesUnknown status
NYP/Weill Cornell Medical Center
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New YorkUnited StatesUnknown status
University of Oklahoma Health Sciences Center
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Oklahoma CityUnited StatesUnknown status
UC Irvine Health- Chao Family Comprehensive Cancer Center
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OrangeUnited StatesUnknown status
University of Chicago Medicine-Orland Park
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Orland ParkUnited StatesUnknown status
Mayo Clinic Hospital in Arizona
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PhoenixUnited StatesUnknown status
UPMC Hillman Cancer Center
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PittsburghUnited StatesUnknown status
VCU Massey Comprehensive Cancer Center
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RichmondUnited StatesUnknown status
University of California Davis Comprehensive Cancer Center
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SacramentoUnited StatesUnknown status
Huntsman Cancer Institute/University of Utah
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Salt Lake CityUnited StatesUnknown status
Montefiore Medical Center - Moses Campus
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The BronxUnited StatesUnknown status
Montefiore Medical Center-Einstein Campus
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The BronxUnited StatesUnknown status
Montefiore Medical Center-Weiler Hospital
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The BronxUnited StatesUnknown status
University of Cincinnati Cancer Center-West Chester
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West ChesterUnited StatesUnknown status
University of Kansas Hospital-Westwood Cancer Center
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WestwoodUnited StatesUnknown status
Wake Forest University Health Sciences
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Winston-SalemUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.