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NCT Number: NCT07451912

Venetoclax Plus Hypomethylating Agents and Subcutaneous Cytarabine for CEBPA-Mutated AML

The goal of this clinical trial is to learn if a treatment combination-venetoclax plus hypomethylating agents (like azacitidine or decitabine) and low-dose cytarabine-works to treat adults with newly diagnosed CEBPA-mutated acute myeloid leukemia (AML) who can't tolerate intensive chemotherapy. It will also check how safe this treatment combination is and explore how the disease might change if it comes back. The main questions it aims to answer are:

1. How well does this treatment combination prevent the disease from coming back (relapse-free survival)? 2. What percentage of participants achieve a good response (complete remission or complete remission with incomplete blood cell recovery) after 2 treatment cycles? 3. What percentage of participants have no detectable remaining leukemia cells (measurable residual disease, MRD) after treatment? What side effects do participants have, and how serious are these side effects?

Participants will:

1. First, go through a 2-cycle "induction phase": Take venetoclax by mouth (100mg on day 1, 200mg on day 2, 400mg from day 3 to day 28), get hypomethylating agents (azacitidine injected under the skin or decitabine injected into a vein), and low-dose cytarabine (injected under the skin) as planned. 2. If they respond well to induction treatment, move to a "consolidation phase" and receive at least 4 more cycles of the same treatment combination. 3. Have regular check-ups during treatment (like blood tests, bone marrow tests, and heart checks) to monitor treatment response and side effects. 4. Be followed up for 2 years after treatment ends to check if the disease comes back and their overall health.

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Key information

Conditions

Acute Myeloid Leukemia (AML) CEBPA Mutation Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Neoplasms Neoplasms by Histologic Type Unfit, New-diagnosis AML

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location status: Recruiting

Location contact

The First Affiliated Hospital of Soochow University

CONTACT

[email protected]

0086-0512-67972861

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients can participate in this study only if they meet all the following enrollment criteria:
  • Aged over 18 years, male, or female who is neither pregnant nor lactating;
  • Newly diagnosed acute myeloid leukemia (AML) with CEBPA mutation;
  • ECOG performance status ≤ Grade 3;
  • Capable of understanding and willing to participate in the study, and able to sign the informed consent form;
  • Patients are judged as "unfit" according to the Ferrara criteria. A patient is considered "unfit" if they meet at least one of the following criteria:
  • Advanced age: > 75 years old;
  • Presence of severe underlying comorbidities involving the heart, lungs, kidneys, or liver; ③ Presence of active infection unresponsive to anti-infective treatment;
  • Presence of cognitive impairment; ⑤ Poor performance status (persistent ECOG score ≥ Grade 3); ⑥ Other comorbidities judged by the investigator to make the patient unfit for intensive chemotherapy.

Exclusion criteria

  • Patients are not suitable for participating in this study if they meet any of the following exclusion criteria:
  • Pregnant or lactating women;
  • Previous receipt of chemotherapy or targeted drug therapy for leukemia before the study treatment (except oral hydroxyurea used to reduce white blood cell count and/or leukapheresis);
  • As known to the participant and the investigator, the participant may be unable to complete all study visits or procedures required by the study protocol (including follow-up visits) and/or unable to comply with the required study procedures;
  • Other conditions judged by the investigator to make the patient unfit for participating in this study.

Treatment and study plan

Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients)

Drug
  • Induction Therapy (2 Cycles in Total) All medications are administered in a 28-day cycle; the second cycle is given regardless of the response to the first cycle.

Medication Generic Name Dosage Form Dosage Administration Frequency Duration per Cycle Venetoclax Oral tablets 100 mg on Day 1; 200 mg on Day 2; 400 mg from Day 3 to Day 28 Once daily (oral) Days 1-28 Azacitidine (alternative to decitabine) Injectable 75 mg/m² per day Once daily (subcutaneous injection) Days 1-7 Decitabine (alternative to azacitidine) Injectable 20 mg/m² per day Once daily (intravenous injection) Days 1-5 Cytarabine (low-dose) Injectable 20 mg/m² per day Once daily (subcutaneous injection) Days 1-10

  • Consolidation Therapy (At Least 4 Cycles) Same as the therapy of induction

Primary outcomes

  1. Relapse-Free Survival (RFS)

    Time frame: From date of achieving CR/CRi until first occurrence of disease relapse or death from any cause, assessed up to 48 months (2 years after the last patient has been enrolled into the study).

    Defined as the time from the date of achieving complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) to the first occurrence of disease relapse (reappearance of ≥5% blasts in the bone marrow, presence of blasts in the peripheral blood, or new extramedullary disease) or death from any cause, whichever comes first. For participants who remain in remission at the end of the follow-up period, RFS is censored at the date of the last confirmed remission assessment.

Secondary outcomes

  1. Composite Remission Rate after 2 Induction Cycles

    Time frame: Proportion of participants who achieve CR or CRi after completing 2 cycles of induction therapy. (Each cycle is 28 days)

    Proportion of participants who achieve CR or CRi after completing 2 cycles of induction therapy. (Each cycle is 28 days) CR: Defined by the following criteria simultaneously: bone marrow blasts <5%, no blasts in the peripheral blood, absolute neutrophil count >1.0×10⁹/L, platelet count >100×10⁹/L, no extramedullary infiltration, and no relapse within 4 weeks.

    CRi: Meets all CR criteria except for incomplete hematologic recovery (absolute neutrophil count ≤1.0×10⁹/L or platelet count ≤100×10⁹/L).

  2. Minimal Residual Disease (MRD) Negativity Rate

    Time frame: 21 to 28 days after the end of every treatment

    Proportion of participants with MRD <1×10-⁴ detected by flow cytometry after treatment.

  3. Duration of Remission (DOR)

    Time frame: From date of achieving CR/CRi until first occurrence of disease relapse or death from any cause, assessed up to 60 months.

    If no relapse or death occurs during follow-up, DOR is censored at the last follow-up date.

  4. Relapse Rate

    Time frame: Proportion of participants who experience disease relapse after achieving CR/CRi, assessed up to 48 months (2 years after the last patient has been enrolled into the study).

    Proportion of participants who experience disease relapse after achieving CR/CRi during the study period.

  5. Overall Survival (OS)

    Time frame: From start of treatment until death from any cause, assessed up to 48 months (2 years after the last patient has been enrolled into the study).

    Time from the start of treatment to death from any cause. For participants who remain alive at the end of the follow-up period, OS is censored at the date of the last survival confirmation.

Study contacts

Contact information is provided by the study sponsor or research team.

Suning Chen

CONTACT

[email protected]

008613814881746

Wenzhi Cai

CONTACT

008615106202752

Sponsors and collaborators

Lead sponsor

The First Affiliated Hospital of Soochow University

Other

Registry information

Official study title

Prospective Multicenter Clinical Study of Venetoclax Combined With Hypomethylating Agents and Subcutaneous Cytarabine in Induction Therapy for CEBPA-Mutated Acute Myeloid Leukemia

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Mar 5, 2026
Registry last updated
Mar 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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