Venetoclax with hypomethylating drugs and low-dose cytarabine for adults with CEBPA‑mutated AML who can't tolerate intensive chemotherapy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 1/2, single‑center study gives adults with newly diagnosed CEBPA‑mutated AML who are judged unfit for intensive chemotherapy two cycles of induction therapy combining oral venetoclax with a hypomethylating agent (azacitidine or decitabine) plus subcutaneous low‑dose cytarabine. The phase 1 portion focuses on establishing safe dosing and tolerability, and the phase 2 portion expands to measure efficacy signals. Primary outcomes include relapse‑free survival, rate of complete remission or complete remission with incomplete blood count recovery (CR/CRi) after two cycles, MRD negativity, and treatment‑related adverse events. The protocol also includes exploratory analyses to track how the disease may evolve at relapse.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Patients can participate in this study only if they meet all the following enrollment 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 to sign the informed consent form;
  5. 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:

  1. Pregnant or lactating women;
  2. 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);
  3. 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;
  4. Other conditions judged by the investigator to make the patient unfit for participating in this study.

Where it's running

• The First Affiliated Hospital of Soochow University
  Suzhou, Jiangsu, China Recruiting

Study contacts

• Study coordinator: Suning Chen
• Email: chensuning@sina.com
• Phone: 008613814881746

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.