Venetoclax with two other drugs for newly diagnosed CEBPA-mutated acute myeloid leukemia
Tests venetoclax with two other drugs in adults who cannot have intensive chemotherapy.
Plain-language title generated from the registry record
Registry title: Venetoclax Plus Hypomethylating Agents and Subcutaneous Cytarabine for CEBPA-Mutated AML
Key facts
- Where
- Suzhou, ChinaSee the site
- Phase
- Phase 1/2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with newly diagnosed acute myeloid leukemia (AML) with a CEBPA mutation who cannot have intensive chemotherapy.
- Age
- Over 18 years
- Sex
- Men and women; women must not be pregnant or breastfeeding
- Disease stage
- Newly diagnosed AML with a CEBPA mutation, judged 'unfit' for intensive chemotherapy by the Ferrara criteria (for example older than 75, severe heart, lung, kidney or liver disease, an infection that does not respond to treatment, cognitive impairment, or ECOG score of 3 or more). ECOG performance status must be 3 or lower.
- Earlier treatment
- No earlier chemotherapy or targeted drug treatment for leukemia. Oral hydroxyurea or leukapheresis to lower white blood cell count is allowed.
- Biomarker or test result
- CEBPA mutation
Main reasons for not taking part
- Pregnant or breastfeeding women
- Earlier chemotherapy or targeted drug treatment for leukemia
- Unable to complete all study visits and procedures, including follow-up
- Other conditions that the investigator judges make joining the trial unsafe or unsuitable
Read by machine on 13 November 2026.
Where in China
- The First Affiliated Hospital of Soochow UniversitySuzhou, ChinaContact: The First Affiliated Hospital of Soochow UniversityTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- The First Affiliated Hospital of Soochow University
- Trial code
- NCT07451912
- Record updated
- 5 March 2026
- Original description
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:Read full original descriptionShow less
Prospective Multicenter Clinical Study of Venetoclax Combined With Hypomethylating Agents and Subcutaneous Cytarabine in Induction Therapy for CEBPA-Mutated Acute Myeloid Leukemia
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.
Inclusion: 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: 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.