Actively Recruiting
Targeting Measurable Residual Disease in Acute Myeloid Leukemia: A Phase 1/2 Study of Tagraxofusp, Azacitidine, and Venetoclax
Led by Jacqueline Garcia, MD · Updated on 2026-02-05
31
Participants Needed
2
Research Sites
104 weeks
Total Duration
On this page
Sponsors
J
Jacqueline Garcia, MD
Lead Sponsor
S
Stemline Therapeutics, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating a new combination of three drugs—azacitidine, venetoclax, and tagraxofusp—to treat patients with Acute Myeloid Leukemia (AML) who have leftover leukemia cells that cannot be seen with the naked eye. This Phase 1/2 clinical trial aims to assess the safety and how well this drug combination controls residual AML and prevents the disease from coming back. The study builds on FDA approvals of venetoclax and azacitidine together for AML and tagraxofusp alone for another leukemia type, but this combination is not yet FDA-approved for AML treatment. The study involves two groups of participants. In Phase 1, up to 12 people receive escalating doses of tagraxofusp combined with fixed doses of azacitidine and venetoclax to find the safest and best dose. Treatment cycles last 28 days, with azacitidine given daily for seven days, tagraxofusp infused on days 4 to 6, and venetoclax taken on days 1 and 14. In Phase 2, 19 participants receive the recommended dose of tagraxofusp plus azacitidine and venetoclax with the same schedule. Bone marrow biopsies and aspirations occur regularly during treatment to monitor response. After treatment, participants are followed for up to two years. Participants will have regular visits including blood tests, imaging scans like CT, MRI, or PET, heart function tests, and bone marrow examinations. Researchers will monitor for side effects, measure disease remission, and check for minimal residual disease to evaluate treatment impact. The study expects to last about four years with around 31 participants. Outcomes such as remission duration, survival, relapse rates, and safety events will be assessed during treatment and follow-up.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosed with Acute Myeloid Leukemia (including de novo, secondary, or AML from MDS)
- In complete remission (CR, CRi, or CRh) with less than 5% leukemia cells in bone marrow
- Evidence of CD123 positive cells by central assessment
- Measurable disease of at least 0.1% by flow cytometry
- ECOG performance status of 0 to 2
- Adequate organ and marrow function as defined by specific laboratory limits
- Albumin level of at least 3.2 g/dL
- Left ventricular ejection fraction at or above institutional lower limit within 30 days before treatment
- Negative pregnancy test for women of childbearing potential and agreement to use contraception
- Willing and able to follow study visit schedules and protocol requirements
You will not qualify if you...
- Prior treatment with CD123-targeted therapy
- Diagnosis of acute promyelocytic leukemia
- Received intensive chemotherapy within 2 weeks before study start or venetoclax within 5 days
- Imminent stem cell transplant planned
- History of prior allogeneic stem cell transplant
- Uncontrolled serious lung disease
- Past severe capillary leak syndrome
- Active hepatitis B or C with detectable viral load
- Known HIV infection with detectable viral load
- Concurrent or recent malignancy within 6 months except certain treated cancers
- Uncontrolled fungal, bacterial, or viral infections
- Severe heart disease with NYHA Class III or IV symptoms
- Evidence of ongoing alcohol or drug abuse
- Active or symptomatic central nervous system involvement
- Use of moderate or strong CYP3A inducers within 7 days before treatment
- Consumption of grapefruit, Seville oranges, or star fruit within 3 days before treatment
- Pregnant or breastfeeding women due to potential risks to baby
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 2 years
Participants receive a combination of three drugs: azacitidine, venetoclax, and tagraxofusp in 28-day cycles. During each cycle, azacitidine is given daily for 7 days, tagraxofusp is given daily for 3 days, and venetoclax is given on days 1 and 14. Bone marrow biopsies and aspirations are performed regularly to monitor disease.
Baseline visit plus repeated visits for treatment cycles every 28 days, including bone marrow biopsies on Day 28 of Cycle 1, Day 1 of Cycles 4, 6, 9, 12, and every 3 cycles thereafter, and an end of treatment visit
Duration - Up to 2 years
Participants are followed for up to 2 years after completing treatment to monitor survival status and disease relapse.
Visits every 12 weeks for survival assessment
Trial Site Locations
Total: 2 locations
1
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
2
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
Research Team
J
Jacqueline Garcia, MD
J
Jacqueline Garcia, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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