NCT06429098

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: The First Affiliated Hospital of Soochow University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
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

Timeline

• Start date: 2024-01-01
• Primary completion: 2027-06-30
• Completion: 2027-12-31
• First posted: 2024-05-24
• Last updated: 2025-09-04

Locations

2 sites across 1 country: China