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

Last updated: March 4, 2026
Sponsor: The First Affiliated Hospital of Soochow University
Overall Status: Active - Recruiting

Phase

1/2

Condition

Leukemia (Pediatric)

Treatment

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

Clinical Study ID

NCT07451912
SZAML-05
  • Ages > 18
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients can participate in this study only if they meet all the followingenrollment criteria:

  1. Aged over 18 years, male, or female who is neither pregnant nor lactating;

  2. Newly diagnosed acute myeloid leukemia (AML) with CEBPA mutation;

  3. ECOG performance status ≤ Grade 3;

  4. Capable of understanding and willing to participate in the study, and able tosign the informed consent form;

  5. Patients are judged as "unfit" according to the Ferrara criteria. A patient isconsidered "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 unresponsiveto anti-infective treatment;
  • Presence of cognitive impairment; ⑤ Poor performance status (persistent ECOG score ≥ Grade 3); ⑥ Other comorbidities judgedby the investigator to make the patient unfit for intensivechemotherapy.

Exclusion

Exclusion Criteria:

  • Patients are not suitable for participating in this study if they meet any of thefollowing exclusion criteria:

  1. Pregnant or lactating women;

  2. Previous receipt of chemotherapy or targeted drug therapy for leukemia beforethe study treatment (except oral hydroxyurea used to reduce white blood cellcount and/or leukapheresis);

  3. As known to the participant and the investigator, the participant may be unableto complete all study visits or procedures required by the study protocol (including follow-up visits) and/or unable to comply with the required studyprocedures;

  4. Other conditions judged by the investigator to make the patient unfit forparticipating in this study.

Study Design

Total Participants: 29
Treatment Group(s): 1
Primary Treatment: Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients)
Phase: 1/2
Study Start date:
September 15, 2025
Estimated Completion Date:
December 31, 2027

Connect with a study center

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu 215006
    China

    Active - Recruiting

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