Actively Recruiting

Phase 1
Phase 2
Age: 18Years +
All Genders
NCT05010122

ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Led by M.D. Anderson Cancer Center · Updated on 2026-02-18

42

Participants Needed

1

Research Sites

342 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This phase I/II trial studies the best dose of gilteritinib given together with ASTX727 and venetoclax and the effect of ASTX727, venetoclax, and gilteritinib in treating patients with FLT3-mutated acute myeloid leukemia that is newly diagnosed, has come back (relapsed) or does not respond to treatment (refractory) or high-risk myelodysplastic syndrome. Chemotherapy drugs, such as ASTX727, 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 ASTX727, venetoclax, and gilteritinib may help to control the disease.

CONDITIONS

Official Title

ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adults aged 18 years or older with relapsed or refractory FLT3-mutated AML or intermediate-2 or high-risk MDS (Phase I)
  • Adults aged 18 years or older with newly diagnosed FLT3-mutated AML who are ineligible for standard induction therapy due to age or comorbidities (Phase II cohort A)
  • Adults aged 18 years or older with relapsed or refractory FLT3-mutated AML or intermediate-2 or high-risk MDS who have received one prior therapy (Phase II cohort B)
  • Patients with FLT3-ITD or FLT3 D835 mutations
  • ECOG performance status of 0 to 3
  • Total serum bilirubin less than or equal to 2.5 times the upper limit of normal unless due to specific conditions approved by the investigator
  • ALT or AST less than or equal to 3 times the upper limit of normal unless due to leukemia approved by the investigator
  • Creatinine clearance of 30 mL/min or higher
  • Ability to swallow medication
  • Signed informed consent
  • Patients may use hydroxyurea or one dose of cytarabine to reduce white blood cell count below 25 x 10^9/L before starting treatment
Not Eligible

You will not qualify if you...

  • Patients in Phase II cohort A who have received prior therapy for AML (except certain allowed treatments)
  • Patients in Phase II cohort B with three or more prior lines of therapy
  • Patients suitable for and willing to receive intensive induction chemotherapy (Phase II cohort A only)
  • Congenital long QT syndrome or corrected QT interval greater than 450 msec
  • Active serious infection not controlled by antibiotics
  • Active grade III-V cardiac failure
  • Active central nervous system leukemia
  • Known active hepatitis B or C infection or positive hepatitis B surface antigen
  • Concurrent malignancy that may interfere with safety or efficacy assessments without investigator approval
  • Recent use of strong CYP3A or p-glycoprotein inducers within 3 days prior to enrollment
  • Use of investigational antileukemic or chemotherapy agents within 7 days before study entry unless fully recovered
  • Pregnant or breastfeeding women; women of childbearing potential must have a negative pregnancy test and use effective contraception; men must use contraception
  • Medical, psychiatric, cognitive, or other conditions preventing understanding or compliance with the study protocol

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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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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