Comparing two treatments for newly diagnosed high-risk acute myeloid leukemia

Study of the Efficacy and Safety of Venetoclax Plus Azacytidine Versus Daunorubicin Plus Cytarabine in Adult Acute Myeloid Leukemia (AML) Patients With Adverse Risk Features

This study is testing whether a new combination of medications can work better than the standard treatment for adults with newly diagnosed high-risk acute myeloid leukemia.

Quick facts

• Phase: Phase2; Phase3
• Study type: Interventional
• Enrollment: 116 (estimated)
• Ages: 18 Years to 64 Years
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 2 sites (Suzhou, Jiangsu and 1 other locations)
• Trial ID: NCT05939180 on ClinicalTrials.gov

What this trial studies

This open-label, multicenter study aims to compare the efficacy and safety of venetoclax plus azacytidine against the conventional daunorubicin plus cytarabine regimen in adults with newly diagnosed acute myeloid leukemia (AML) exhibiting adverse risk features. Participants will be randomly assigned to one of the two treatment groups and will receive induction therapy followed by consolidation chemotherapy and potential stem cell transplantation upon achieving remission. The study also incorporates a novel targeted sequencing panel for rapid screening of adverse mutations. Follow-up will occur every 1 to 2 months for up to 2 years post-treatment.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-64 with newly diagnosed AML and adverse risk features according to the 2022 European Leukemia Net risk stratification.

Not a fit: Patients with acute promyelocytic leukemia, central nervous system infiltration, or those with myeloid sarcoma will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a more effective treatment option for patients with high-risk AML, potentially improving remission rates and overall survival.

How similar studies have performed: Other studies have shown promising results with similar targeted therapies in AML, indicating potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. Gender: female or male.
2. Age:18-64 years old.
3. Patients with newly diagnosed AML according to the WHO 2022 classification.
4. AML patients with adverse risk features according to the 2022 European Leukemia Net risk stratification.
5. Untreated AML (hydroxyurea, and low dose cytarabine with cummulative dose \<1.0g are permitted).
6. ECOG: 0-2.
7. Adequate liver function: Total bilirubin ≤ 1.5×upper limit of normal (ULN); aspartate aminotransferase (AST) ≤3×ULN (liver infiltration of leukemia: ≤5×ULN); alanine aminotransferase (ALT)≤3×ULN (liver infiltration of leukemia: ≤5×ULN) .
8. Adequate Renal function: Ccr (Creatinine Clearance Rate) ≥30 ml/min.
9. Be able to understand and be willing to participate in the study. Be able to provide written informed consent.

Exclusion Criteria:

1. Patients with acute promyeloid leukemia.
2. AML with central nervous system infiltration.
3. Patients diagnosed with myeloid sarcoma.
4. Patients have AML secondary to MDS and previously been treated with hypomethylating agents.
5. Patients with active infection, which is considered as uncontrollable by the investigator.
6. Patients with active hepatitis B, hepatitis C and HIV infection.
7. Patients with heart failure (grade 3-4);
8. Patients who are pregnant or breastfeeding.
9. Patients who refused to be enrolled in the study. Patients who are considered as ineligible for the enrollment by the investigators.

Where this trial is running

Suzhou, Jiangsu and 1 other locations

• The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology — Suzhou, Jiangsu, China (Recruiting)
• Ethical Committee of the First Affliated Hospital of Soochow University — Suzhou, Jiangsu, China (Recruiting)

Study contacts

• Principal investigator: Su-ning Chen — The First Affiliated Hospital of Soochow University
• Study coordinator: Su-ning Chen

Conditions Acute Myeloid Leukemia; Adverse risk, newly diagnosed, induction therapy