Optimizing AML treatment using early peripheral blood minimal residual disease

A Multicenter, Open-label, Prospective, Randomized Controlled Study to Optimize the Treatment of Patients With Acute Myeloid Leukemia Based on Early Peripheral Blood Minimal Residual Disease

This trial tests whether adding venetoclax based on early peripheral blood minimal residual disease can help adults with newly diagnosed AML who have a suboptimal early response to chemotherapy.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 70 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: The First Hospital of Jilin University (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Changchun, Jilin)
• Trial ID: NCT07023588 on ClinicalTrials.gov

What this trial studies

This is a prospective, open-label, randomized controlled Phase 2 trial led by The First Hospital of Jilin University that enrolls adults with newly diagnosed AML who have Day 6 peripheral blood blasts meeting the protocol threshold. Eligible participants are randomized to receive standard induction chemotherapy with or without the addition of venetoclax, with approximately 70 subjects planned (35 per arm). The study collects baseline demographics, disease and treatment details, safety data, and outcome measures including response and relapse, and performs statistical comparisons of efficacy and safety between arms. The protocol uses early peripheral blood minimal residual disease (MRD) measurements to select patients with suboptimal early response for the intervention.

Who should consider this trial

Good fit: Ideal candidates are adults (≥18 years) with newly diagnosed AML confirmed by bone marrow testing, ECOG 0–2, Day 6 peripheral blood blast percentage ≥1%, adequate kidney and liver function, judged suitable for DA/IA chemotherapy, and able to give informed consent.

Not a fit: Patients with acute promyelocytic leukemia, mixed phenotype acute leukemia, known CNS involvement, significant organ dysfunction, or those without early MRD signals indicating suboptimal response are unlikely to benefit from this MRD-guided venetoclax approach.

Why it matters

Potential benefit: If successful, adding venetoclax guided by early peripheral blood MRD could deepen remissions and lower relapse risk by personalizing early therapy for patients with suboptimal initial chemotherapy response.

How similar studies have performed: Venetoclax has shown promising responses in AML when combined with other agents, but using early peripheral blood MRD to guide the addition of venetoclax is a relatively new strategy with limited direct evidence to date.

Eligibility criteria

Inclusion Criteria:

1. Newly diagnosed AML patients confirmed by bone marrow morphology and immunophenotyping.
2. Complete MICM (Morphology, Immunophenotyping, Cytogenetics, Molecular genetics) work-up.
3. Age ≥ 18 years.
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
5. Day 6 Peripheral Blood Blast Percentage (D6PBBP) ≥ 1%.
6. Renal function with creatinine clearance ≥ 50 mL/min (calculated using the Cockcroft-Gault formula or measured by 24-hour urine collection).
7. Liver function with Aspartate Aminotransferase (AST) ≤ 2.5 × ULN\*; Alanine Aminotransferase (ALT) ≤ 2.5 × ULN\*; Total bilirubin ≤ 1.5 × ULN\* (\*Unless considered due to leukemia infiltration).
8. Patients deemed suitable to receive DA/IA therapy by the investigator.
9. Signed informed consent form. -

Exclusion Criteria:

1. Acute promyelocytic leukemia (APL).
2. Mixed phenotype acute leukemia (MPAL).
3. AML patients with known central nervous system (CNS) involvement.
4. Presence of extramedullary disease (EMD).
5. Significant hepatic or renal dysfunction exceeding the inclusion criteria limits.
6. Severe cardiac disease, including congestive heart failure, myocardial infarction, or cardiac dysfunction.
7. Concurrent active malignancy of other organ systems (patients with a history of cured malignancy may be eligible).
8. Active tuberculosis or HIV-positive patients.
9. Concurrent other hematological disorders.
10. Pregnant or lactating women.
11. Inability to comprehend or comply with the study protocol.
12. Hypersensitivity to any component of the drugs involved in the protocol.
13. Inability to take oral medication or patients with malabsorption syndrome.
14. Uncontrolled systemic infection.
15. Prior venetoclax treatment and/or current participation in any other study involving investigational agents.

Where this trial is running

Changchun, Jilin

• The First Hospital of Jilin University — Changchun, Jilin, China (Recruiting)

Study contacts

• Study coordinator: Yehui Tan

Conditions Acute Myeloid Leukemia