Venetoclax plus CACAG versus 3+7 for acute monocytic leukemia

Venetoclax Combined With CACAG Regimen Versus "3+7" Regimen in the Treatment of Acute Monocytic Leukemia: A Prospective, Randomized, Controlled Study

This trial will test whether adding venetoclax to a CACAG combination works better than standard 3+7 chemotherapy for people aged 14–60 with acute monocytic leukemia.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 204 (estimated)
• Ages: 14 Years to 60 Years
• Sex: All
• Sponsor: Chinese PLA General Hospital (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 3 sites (Beijing and 2 other locations)
• Trial ID: NCT07512700 on ClinicalTrials.gov

What this trial studies

This is a multicenter, randomized Phase II trial in Beijing that will enroll 204 participants aged 14–60 with acute monocytic leukemia and randomly assign them to either venetoclax combined with the CACAG backbone (azacitidine, cytarabine, aclarubicin, chidamide and G-CSF) or to standard 3+7 induction with daunorubicin and cytarabine. Treatment consists of two cycles over about 8–10 weeks, with participants who do not achieve at least a partial response after the first cycle potentially withdrawn for alternative therapy. Key outcomes include response rates, safety/tolerability, and overall clinical outcomes during the study period. The trial compares a modern targeted-agent combination against the historical standard induction regimen to see if the new approach improves remission and safety profiles.

Who should consider this trial

Good fit: Ideal candidates are people aged 14–60 with newly diagnosed acute monocytic leukemia per the 2016 WHO criteria (excluding APL), who can give informed consent, have ECOG 0–3, adequate liver and kidney function, and no uncontrolled infection or pregnancy.

Not a fit: Patients unlikely to benefit include those with acute promyelocytic leukemia, active uncontrolled infection, significant organ dysfunction, pregnancy/lactation, or known hypersensitivity to study drugs.

Why it matters

Potential benefit: If successful, the venetoclax plus CACAG approach could produce higher remission rates or a more favorable safety profile than standard 3+7 induction for acute monocytic leukemia.

How similar studies have performed: Venetoclax combined with hypomethylating agents and other backbones has shown promising response rates in AML overall, but evidence specifically in acute monocytic leukemia is more limited and results have been mixed.

Eligibility criteria

Inclusion Criteria:

* Voluntary participation with written informed consent signed by the participant or a legal guardian; willingness to comply with all study procedures.
* Age 14 to 60 years at screening, no gender restriction.
* Diagnosis of acute monocytic leukemia according to the 2016 WHO classification, excluding acute promyelocytic leukemia.
* No history of severe allergic reactions.
* Liver function: ALT and AST ≤ 2.5 × upper limit of normal (ULN); total bilirubin ≤ 2 × ULN.
* Renal function: serum creatinine ≤ 1.5 × ULN
* No uncontrolled infection or severe psychiatric disorder.
* ECOG performance status 0-3; life expectancy ≥ 4 months.

Exclusion Criteria:

* Known hypersensitivity or contraindication to any study drug.
* Pregnancy or lactation.
* Active infection.
* Long-term smoking or alcohol abuse that may interfere with study outcome evaluation.
* Psychiatric illness or other condition that prevents informed consent or compliance with study procedures.
* Major organ surgery within 6 weeks prior to enrollment.
* Abnormal liver function: total bilirubin \> 2× ULN, ALT/AST \> 2.5 × ULN; abnormal renal function: serum creatinine \> 1.5 × ULN.
* Any condition deemed unsuitable for the study by the investigator (e.g., poor compliance, substance abuse).

Where this trial is running

Beijing and 2 other locations

• Air Force Medical Center, PLA — Beijing, China (Recruiting)
• Chinese PLA General Hospital — Beijing, China (Recruiting)
• PLA Strategic Support Force's Characteristic Medical Center — Beijing, China (Recruiting)

Study contacts

• Principal investigator: Liping Dou, Doctor — Chinese PLA General Hospital
• Study coordinator: Liping Dou, Doctor

Conditions Acute Monocytic Leukemia; Venetoclax+CACAG