Study of A Venetoclax-based, Anthracycline-free Regimen in Newly Diagnosed CBFβ::MYH11(+) AML
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Venetoclax, azacitidine, decitabine, Cytarabine.
- Who it may be relevant to
- Registry conditions: Acute Myeloid Leukemia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Study of A Venetoclax-based, Anthracycline-free Regimen in Patients With Newly Diagnosed CBFβ::MYH11-positive Acute Myeloid Leukemia
Overview
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.
Detailed description
Primary Objectives:
To determine the CR (complete remission) / CRi (complete remission with incomplete blood count recovery) rate of 2 cycles of VEN/HMA in patients with newly diagnosed (ND) CBFβ::MYH11-positive acute myeloid leukemia(AML).
Secondary Objectives:
1. To determine the overall response rate (ORR) of 2 cycles of VEN/HMA in patients with ND CBFβ::MYH11-positive AML. 2. To determine the safety of the combination regimen. 3. To study the trajectories of molecular measurable residual disease (MRD) during the therapy. 4. To evaluate the impact of baseline genomic alterations on response and survival of the combination regimen. 5. To assess the duration of response, overall survival (OS) and event free survival (EFS) of patients.
OUTLINE:
INDUCTION:
Patients with newly diagnosed CBFβ::MYH11(+) AML 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:
Patient fitness will be reassessed according to the Ferrara criteria if CR or CRi is achieved after 2 cycles of VEN/HMA. 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.
After completion of study treatment, patients are followed up at 30 days and then every 3 months thereafter.
Interventions
- Drug Venetoclax
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. - Drug azacitidine
Given SC - Drug decitabine
Given IV - Drug Cytarabine
Given IV
Primary outcome measures
- composite complete remission rate [Time frame: after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).]
Secondary outcome measures (7)
- Overall response rate (ORR) [Time frame: after 2 cycles of induction therapy with VEN/HMA (each cycle is 28 days).]
- Incidence of adverse events [Time frame: From the start of treatment until death or last follow-up, assessed for up to 3 years.]
- measurable residual disease (MRD) negativity [Time frame: From the start of treatment until death or last follow-up, assessed for up to 3 years.]
- Impact of concurrent gene mutations [Time frame: Baseline]
- Overall survival (OS) [Time frame: From the start of treatment until death or last follow-up, assessed for up to 3 years.]
- Event-free survival (EFS) [Time frame: up to 3 years.]
- Duration of response (DOR) [Time frame: up to 3 years.]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 2 centers
- Ethical Committee of the First Affiliated Hospital of Soochow University — Suzhou
- The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology — Suzhou
Identifiers
NCT: NCT06429098 · 2023371