Actively Recruiting

Phase 1
Phase 2
Age: 18Years +
All Genders
NCT04140487

Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm

Led by M.D. Anderson Cancer Center · Updated on 2026-04-16

97

Participants Needed

1

Research Sites

454 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This phase I/II trial studies the side effects and best dose of gilteritinib and to see how well it works in combination with azacitidine and venetoclax in treating patients with FLT3-mutation positive acute myeloid leukemia, chronic myelomonocytic leukemia, or high-risk myelodysplastic syndrome/myeloproliferative neoplasm that has come back (recurrent) or has not responded to treatment (refractory). Drugs used in chemotherapy, 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. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Gilteritinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine, venetoclax, and gilteritinib may work better compared to azacitidine and venetoclax alone in treating patients with acute myeloid leukemia, chronic myelomonocytic leukemia, or myelodysplastic syndrome/myeloproliferative neoplasm.

CONDITIONS

Official Title

Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adults 18 years or older with relapsed or refractory FLT3-mutated AML, CMML, or intermediate-2/high-risk MDS/MPN (Phase I cohort)
  • Adults 18 years or older with newly diagnosed FLT3-mutated AML (Phase II cohort A)
  • Adults 18 years or older with relapsed or refractory FLT3-mutated AML, CMML, or intermediate-2/high-risk MDS/MPN (Phase II cohort B)
  • Patients with FLT3-internal tandem duplication or FLT3 D835 mutations
  • Eastern Cooperative Oncology Group (ECOG) performance status of 3 or less
  • Total serum bilirubin 2.5 times the upper limit of normal or less, unless due to certain approved conditions
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) three times the upper limit of normal or less, unless due to underlying leukemia approved by the principal investigator
  • Creatinine clearance 30 mL/min or higher
  • Ability to swallow medications
  • Signed informed consent
Not Eligible

You will not qualify if you...

  • Patients suitable for and willing to receive intensive induction chemotherapy (Phase II cohort A only)
  • Congenital long QT syndrome or corrected QT interval by Fridericia (QTcF) greater than 450 milliseconds, unless manually confirmed to be 450 milliseconds or less
  • Active serious infection not controlled by antibiotics
  • Active grade III-V heart failure as defined by New York Heart Association criteria
  • Active central nervous system leukemia
  • Known HIV positive status
  • Known hepatitis B surface antigen positive or known/suspected active hepatitis C infection unless viral loads are undetectable
  • Use of strong CYP3A or p-glycoprotein inducers within 3 days before enrollment
  • Treatment with investigational antileukemic or chemotherapy agents in the last 7 days before study entry unless fully recovered or rapidly progressive disease
  • Pregnant or breastfeeding women; women of childbearing potential must have a negative pregnancy test and agree to contraception during and after the study; men must agree to contraception during and after the study
  • Prior or concurrent malignancy that might interfere with study assessments unless approved by the principal investigator

AI-Screening

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Trial Site Locations

Total: 1 location

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

N

Nicholas Short

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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