Study of A Venetoclax-based, Anthracycline-free Regimen in Newly Diagnosed CBFβ::MYH11(+) AML

NCT06429098 | Recruiting Phase 2

• 1 other identifier: 2023371
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

Brief Summary

This investigator-initiated, single-arm, phase II trial is aimed to evaluate the efficacy and safety of a venetoclax-based, anthracycline-free regimen in patients with newly diagnosed CBFβ::MYH11-positive acute myeloid leukemia.

Conditions

Acute Myeloid Leukemia

Keywords

venetoclax; acute myeloid leukemia

Outcome Measures

Primary Outcomes (1)

• composite complete remission rate

  CR/CRi rate will be determined.

  after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).

Secondary Outcomes (7)

• Overall response rate (ORR)

  after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).

• Incidence of adverse events

  From the start of treatment until death or last follow-up, assessed for up to 3 years.

• measurable residual disease (MRD) negativity

  From the start of treatment until death or last follow-up, assessed for up to 3 years.

• Impact of concurrent gene mutations

  Baseline

• Overall survival (OS)

  From the start of treatment until death or last follow-up, assessed for up to 3 years.

Study Arms (1)

Venetoclax, Azacitidine/Decitabine/, Cytarabine

EXPERIMENTAL

INDUCTION: Patients receive 2 cycles of VEN/HMA as induction therapy. Venetoclax orally (PO) once daily (QD) on days 1-28, azacitidine subcutaneously (SC) on days 1-7 or decitabine intravenously (IV) over 30-60 minutes on days 1-5. CONSOLIDATION: Fit patients will receive four cycles of consolidation therapy with high-dose cytarabine (2g/m2 every 12 hours, on days 1-3) combined with venetoclax (on days 1-7). Unfit patients will continue to receive VEN/HMA until disease progression.

Drug: Venetoclax; Drug: azacitidine; Drug: decitabine; Drug: Cytarabine

Interventions

Venetoclax DRUG

Given PO, once daily. Treatment in cycle 1, the dose is 100 mg on day 1, then ramp up to 400mg. In all subsequent cycles, the dose of venetoclax is initiated at 400 mg daily.

Also known as: ABT-199, ABT199, Venclexta

Venetoclax, Azacitidine/Decitabine/, Cytarabine

azacitidine DRUG

Given SC

Also known as: 5 AZC, 5-AZC

Venetoclax, Azacitidine/Decitabine/, Cytarabine

decitabine DRUG

Given IV

Also known as: Dacogen

Venetoclax, Azacitidine/Decitabine/, Cytarabine

Cytarabine DRUG

Given IV

Also known as: 1-.beta.-Cytosine arabinoside, Ara-C

Venetoclax, Azacitidine/Decitabine/, Cytarabine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults ≥ 18 years.
• Newly diagnosed CBFβ::MYH11(+) AML.
• Performance status 0-3 on the Eastern Cooperative Oncology Group (ECOG) Scale.
• 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:
• Age\>75 years.
• There are serious underlying heart, lung, kidney, liver complications.
• There are active infections that do not respond to anti-infective therapy.
• There is cognitive impairment.
• Other comorbidities that the doctor determines are not suitable for intensive chemotherapy.

You may not qualify if:

• 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).
• Pregnant or lactating women.
• 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.
• Other conditions deemed unsuitable for participation in this study by the investigator.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (2)

Ethical Committee of the First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215000, China

RECRUITING

The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology

Suzhou, Jiangsu, 215000, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

venetoclax; Azacitidine; Decitabine; Cytarabine; 1-beta-D-arabinofuranosylcytosine 5'-monophosphate

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Arabinonucleosides

Study Officials

• Ying Wang

  The First Affiliated Hospital of Soochow University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ying Wang

CONTACT

008613656214782; wangying77@suda.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 14, 2024
• First Posted: May 24, 2024
• Study Start: January 1, 2024
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: September 4, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)