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

Condition(s) studied

Investigational drug(s) / intervention(s)

Venetoclax + Hypomethylating Agents + Low-Dose Cytarabine for CEBPA-Mutated AML (Unfit Patients): 1. 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 2. Consolidation Therapy (At Least 4 Cycles) Same as the therapy of induction

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

Primary outcome measure(s)

• Relapse-Free Survival (RFS) — 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.

Trial sites (1)

More The First Affiliated Hospital of Soochow University trials in China

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