Venetoclax-based treatment for newly diagnosed acute myeloid leukemia

Study of A Venetoclax-based, Anthracycline-free Regimen in Patients With Newly Diagnosed CBFβ::MYH11-positive Acute Myeloid Leukemia

This study is testing a new treatment using venetoclax for people with newly diagnosed acute myeloid leukemia to see how well it works and how safe it is.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years and up
• 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: NCT06429098 on ClinicalTrials.gov

What this trial studies

This phase II trial evaluates the efficacy and safety of a venetoclax-based, anthracycline-free regimen in patients with newly diagnosed CBFβ::MYH11-positive acute myeloid leukemia (AML). Participants will receive two cycles of venetoclax combined with either azacitidine or decitabine as induction therapy. The study aims to determine the complete remission rate, overall response rate, and safety of the treatment, as well as the impact of genomic alterations on patient outcomes. Patients will be monitored for response duration, overall survival, and event-free survival.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with newly diagnosed CBFβ::MYH11-positive acute myeloid leukemia.

Not a fit: Patients who have previously received treatment for AML or those deemed unfit for intensive chemotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a safer and effective option for patients with a specific type of acute myeloid leukemia.

How similar studies have performed: While this approach is innovative, similar studies have shown promise in using targeted therapies for AML, suggesting potential for success.

Eligibility criteria

Inclusion Criteria:

1. Adults ≥ 18 years.
2. Newly diagnosed CBFβ::MYH11(+) AML.
3. Performance status 0-3 on the Eastern Cooperative Oncology Group (ECOG) Scale.
4. Subject must voluntarily sign and date an informed consent, prior to the initiation of any screening or study-specific procedures.

Ferrara's criteria are used to determine whether a patient is unfit, and a patient is deemed unfit if at least one of the following criteria is met:

1. Age\>75 years.
2. There are serious underlying heart, lung, kidney, liver complications.
3. There are active infections that do not respond to anti-infective therapy.
4. There is cognitive impairment.
5. Other comorbidities that the doctor determines are not suitable for intensive chemotherapy.

Exclusion Criteria:

1. Subject has received treatment with a hypomethylating agent and/or other chemotherapeutic agents either conventional or experimental or targeted drug therapy for AML (except oral hydroxyurea and/or leukocytometry to reduce white blood cell count).
2. Pregnant or lactating women.
3. To the knowledge of the subject and investigator, subject may not be able to complete all study visits or procedures required by the study protocol, including follow-up visits, and/or be unable to comply with the required study procedures.
4. Other conditions deemed unsuitable for participation in this study by the investigator.

Where this trial is running

Suzhou, Jiangsu and 1 other locations

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

Study contacts

• Principal investigator: Ying Wang — The First Affiliated Hospital of Soochow University
• Study coordinator: Ying Wang

Conditions Acute Myeloid Leukemia; venetoclax; acute myeloid leukemia