NCT04128501

Venetoclax and Azacitidine for the Treatment of Acute Myeloid Leukemia in the Post-Transplant Setting

A Phase II Study of Venetoclax in Combination With Azacitidine in the Post-Transplant Setting for AML, T Cell Leukemia, and Mixed Phenotype Acute Leukemia

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 100 (actual)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well venetoclax and azacitidine work for the treatment of acute myeloid leukemia after stem cell transplantation. Venetoclax may stop the growth of cancer cells by blocking BCL-2, a protein needed for cancer cell survival. Chemotherapy drugs, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving venetoclax and azacitidine after a stem cell transplant may help control high risk leukemia and prevent it from coming back after the transplant.

Detailed description

PRIMARY OBJECTIVE: 1\. To determine relapse-free survival after the use of venetoclax in combination with azacitidine given as maintenance therapy or for eradication of minimal residual disease in patients with high risk acute myeloid leukemia (AML) after hematopoietic stem cell transplantation (HSCT). SECONDARY OBJECTIVES: 1. To determine the safety and toxicity of venetoclax in combination with azacitidine (type, frequency, severity of adverse events \[AEs\] and relationship of AEs to venetoclax) 2. To determine response duration, overall survival. 3. To determine Incidence of acute and chronic graft versus host disease (GVHD) 4. To perform matched pairs analysis to obtain bias corrected treatment comparisons of Venetoclax + Vidaza (V+V) to standard therapy in AML patients with no evidence of disease (AML D-) subgroup. EXPLORATORY OBJECTIVE: 1\. To investigate possible relationships between baseline protein and gene expression signatures/mutation profile and BH3 profiling in predicting relapse-free survival time to the combination. This exploratory analysis will be done for the data from the entire study, accounting for the noted biological variables covariates, as well as disease status and whether or not the patient had AML, by fitting a Bayesian time-to-event regression model with RFS the outcome variable. OUTLINE: Patients receiving venetoclax and azacitidine for maintenance after allogeneic stem cell transplantation, receive azacitidine subcutaneously (SC) on days 1-5 and venetoclax orally (PO) once daily (QD) on days 1-7. Patients receiving venetoclax and azacitidine for minimal residual disease after allogeneic stem cell transplant, receive azacitidine SC on days 1-7 and venetoclax PO QD on days 1-14. Treatment repeats every 4-8 weeks for up to 12 cycles in the absence of disease progression or unacceptable toxicity.

Conditions

• Acute Bilineal Leukemia
• Acute Biphenotypic Leukemia
• Acute Myeloid Leukemia
• Mixed Phenotype Acute Leukemia
• T Acute Lymphoblastic Leukemia
• Therapy-Related Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Azacitidine	Given SC
DRUG	Venetoclax	Given PO

Timeline

• Start date: 2020-05-05
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2019-10-16
• Last updated: 2026-03-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study