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OncoMatch/Clinical Trials/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

Is NCT04140487 recruiting? Yes, currently enrolling (May 2026). This Phase 1/2 trial studies multiple treatments including Azacitidine and Gilteritinib for recurrent acute myeloid leukemia.

Phase 1/2RecruitingM.D. Anderson Cancer CenterNCT04140487Data as of May 2026

Treatment: Azacitidine · Gilteritinib · VenetoclaxThis 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.

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Extracted eligibility criteria

Cancer type

Acute Myeloid Leukemia

Myeloproliferative Neoplasm

Biomarker criteria

Required: FLT3 ITD

patients with either FLT3-internal tandem duplication (FLT3-ITD) ... will be eligible

Required: FLT3 D835

patients with ... FLT3 D835 mutations will be eligible

Performance status

ECOG 0–3(Limited self-care)

Prior therapy

Cannot have received: any prior therapy for AML

Exception: Prior therapy for antecedent hematologic disorder is allowed. Prior hydroxyurea or cytarabine given for purposes of cytoreduction is also allowed. Prior all trans-retinoic acid given for presumed acute promyelocytic leukemia is also allowed.

Phase II cohort A: Patients with prior therapy for AML are not eligible.

Lab requirements

Kidney function

Creatinine clearance ≥ 30 mL/min

Liver function

Total serum bilirubin ≤ 2.5 x upper limit of normal (ULN), unless due to Gilbert's syndrome, hemolysis or the underlying leukemia approved by the principal investigator (PI); Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 3 x ULN, unless due to the underlying leukemia approved by the PI

Total serum bilirubin ≤ 2.5 x upper limit of normal (ULN), unless due to Gilbert's syndrome, hemolysis or the underlying leukemia approved by the principal investigator (PI); Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 3 x ULN, unless due to the underlying leukemia approved by the PI; Creatinine clearance ≥ 30 mL/min

Structured fields extracted by AI. May contain errors — verify against the official protocol.

US trial sites

  • M D Anderson Cancer Center · Houston, Texas

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