NCT07148180

A Multi-Site Break Through Cancer Trial: Targeting Measurable Residual Disease in Patients With Acute Myeloid Leukemia: A Phase 1/2 Study of Tagraxofusp, Azacitidine, and Venetoclax

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 31 (estimated)
• Sponsor: Jacqueline Garcia, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this research study is to test the safety and efficacy of a new drug combination with three agents, azacitidine, venetoclax and tagraxofusp. Leftover (residual) leukemia disease that is not visible by eye can be increase the chance of disease recurrence. This research study is to determine if the combination therapy can safely help to control residual Acute Myeloid Leukemia (AML) and to prevent disease recurrence. The names of the study drugs involved in this study are: * Tagraxofusp (a type of CD123-directed cytotoxin) * Azacitidine (a type of standard of care cytidine nucleoside analog) * Venetoclax (a type of standard of care BCL-2 inhibitor)

Detailed description

This phase 1/2 study, single-arm, multi-center, open-labeled clinical trial is to test the safety and efficacy of a new drug combination with three agents, azacitidine, venetoclax and tagraxofusp. Leftover (residual) leukemia disease that is not visible by eye can be increase the chance of disease recurrence. This research study is to determine if the combination therapy can safely help to control residual Acute Myeloid Leukemia (AML) and to prevent disease recurrence. A Phase 1/2 clinical trial tests the safety and effectiveness of an investigational drug combination to learn whether the drug combination works in treating a specific disease. The Phase 1 safety run-in part of the study will determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) for tagraxofusp in combination with Azacitidine and Venetoclax. Phase 2 will test the RP2D for Tagraxofusp in combination with azacitidine and venetoclax. The U.S. Food and Drug Administration (FDA) has approved the doublet combination of venetoclax and azacitidine for the treatment of AML. The U. S. FDA has approved Tagraxofusp monotherapy as a therapy for another type of leukemia called blastic plasmacytoid dendritic cell neoplasm. The U.S. Food and Drug Administration (FDA) has not approved the combination of azacitidine, venetoclax and tagraxofusp as a treatment for AML. The research study procedures include screening for eligibility, in-clinic visits, blood tests, Computerized Tomography (CT) scans, Magnetic Resonance Imaging (MRI) scans, or Positron Emission (PET) scans, X-rays, echocardiograms (ECGs), electrocardiograms (EKGs), and bone marrow biopsies and aspirations. Participation in this research study is expected to last about 4 years. It is expected that about 31 people will take part in this research study. Stemline Therapeutics is supplying the study drug, Tagraxofusp. Break Through Cancer is providing funding to support the laboratory services.

Conditions

• Acute Myeloid Leukaemia (AML)
• Leukemia
• Blood Cancer
• Blood Cancers

Interventions

Type	Name	Description
DRUG	Tagraxofusp	A CD123-directed cytotoxin, Single-use vial, via intravenous (into the vein) infusion per protocol.
DRUG	Azacitidine (AZA)	A cytidine nucleoside analog, single-use vial, via intravenous infusion or subcutaneous (under the skin) injection per standard of care.
DRUG	Venetoclax	A BCL-2 inhibitor, tablet, via orally per standard of care.

Timeline

• Start date: 2026-02-02
• Primary completion: 2028-12-31
• Completion: 2030-12-31
• First posted: 2025-08-29
• Last updated: 2026-02-05

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study