NCT07407660

Shortened Venetoclax Duration Based on Day 14 BM Blasts Versus Standard Therapy in Elderly or Frail Patients With AML Patients Treated With Azacitidine Plus Venetoclax

Shortened Venetoclax Duration Based on Bone Marrow Blasts on Day 14 Versus Standard Therapy in Elderly or Frail Patients With Acute Myeloid Leukemia Treated With Azacitidine Plus Venetoclax: A Multicenter Prospective Randomized Controlled Study

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 250 (estimated)
• Sponsor: Peking University People's Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This study aims to compare the efficacy and safety of a shortened treatment course based on the bone marrow blast count on Day 14 versus standard treatment in patients with acute myeloid leukemia treated with venetoclax plus azacitidine.

Detailed description

The standard 28-day cycle of venetoclax is widely recommended for the first cycle of venetoclax plus azacitidine induction therapy in patients with acute myeloid leukemia (AML). However, it has been found that the 28-day treatment cycle was not tolerant for some patients due to severe myelosuppression and infection, which may lead to treatment interruption and delays in subsequent treatment cycles. This is a multicenter, randomized controlled, open-label, non-inferiority study, which compare the efficacy and safety of a shortened treatment course based on the bone marrow blast count on Day 14 versus standard treatment in AML patients treated with venetoclax plus azacitidine induction therapy. This study plans to enroll 250 newly diagnosed AML patients who are intolerant to intensive chemotherapy regimens. Enrolled subjects will be assigned to either the optimized treatment group or the standard treatment group in a 1:1 ratio with stratified blocked randomization, with ELN 2022 classification as the stratification factor. In the optimized treatment group, if the bone marrow blast count is \<5% on Day 14 of the first induction, the duration of venetoclax will be shortened to 14 days; otherwise, the 28-day course will be completed as scheduled. In the standard treatment group, no bone marrow assessment will be performed on Day 14, and all patients will complete the 28-day treatment course. The duration of venetoclax in the second cycle will be 28 days or 21 days (if complete remission with incomplete hematologic recovery) for the two groups. The primary endpoint is the achievement of complete remission or complete remission with incomplete hematologic recovery (CR/CRi) within 2 treatment courses.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Venetoclax	Tablet
DRUG	Azacitidine (AZA)	Solution for subcutaneous

Timeline

• Start date: 2026-02-01
• Primary completion: 2028-02-01
• Completion: 2030-02-01
• First posted: 2026-02-12
• Last updated: 2026-02-12