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.
PRIMARY OBJECTIVES: I. To establish the maximum tolerated dose (MTD) of the combination of decitabine and cedazuridine (ASTX727), venetoclax and gilteritinib in patients with relapsed/refractory FLT3- mutated acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). (Phase I) II. To determine the complete response (CR)/incomplete hematologic recovery (CRi) rate of the regimen in patients with newly diagnosed or relapsed/refractory FLT3-mutated AML or high-risk MDS. (Phase II) SECONDARY OBJECTIVES: I. To assess other efficacy endpoints (CR rate, minimal residual disease negativity by flow cytometry, relapse-free survival, overall survival). II. To assess proportion of patients proceeding to hematopoietic stem cell transplantation (HSCT). III. To determine the safety of the combination regimen. EXPLORATORY OBJECTIVES: I. To evaluate the impact of baseline genomic alterations on response and survival of the combination regimen. II. To determine the impact of baseline FLT3 allelic ratio on response and survival. III. To evaluate clonal evolution from diagnosis to relapse using single-cell sequencing. IV. To evaluate potential role of minimal residual disease (MRD) detection by sensitive polymerase chain reaction (PCR)/next generation sequencing (NGS) assays for FLT3 mutations. V. To evaluate leukemia stem cell populations over the course of treatment with the combination regimen. VI. To determine the impact of baseline apoptotic protein levels as assessed by mass cytometry (CyTOF) on response and resistance to the regimen. OUTLINE: This is a phase I, dose-escalation study of gilteritinib followed by a phase II study. INDUCTION (CYCLE 1): Patients receive decitabine and cedazuridine orally (PO) once daily (QD) on days 1-5, venetoclax PO QD on days 1-28, and gilteritinib PO QD on days 1-28 in the absence of disease progression or unacceptable toxicity CONSOLIDATION (CYCLES 2-24): Patients receive decitabine and cedazuridine PO QD on days 1-5, gilteritinib PO QD on days 1-28, and venetoclax PO QD on days 1-21. Treatment repeats every 28 days for up to 23 cycles in the absence of disease progression or unacceptable toxicity. MAINTENANCE (CYCLES 24+): Patients receive gilteritinib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days, then every 6 months thereafter.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
42
M D Anderson Cancer Center
Houston, Texas, United States
RECRUITINGMaximum tolerated dose (Phase I)
Time frame: Up to 28 days
Overall response (OR) rate (Phase II)
Will estimate the OR for the combination treatment (defined as the proportion of patients achieving complete response (CR) or incomplete hematologic recovery (CRi) within 2 cycles of treatment), along with the 95% credible interval.
Time frame: Up to 2 cycles of treatment (1 cycle = 28 days)
Complete response rate
Will also be estimated along with 95% credible interval.
Time frame: Up to 2 years
To assess minimal residual disease negativity by flow cytometry
Will also be estimated along with 95% credible interval.
Time frame: Up to 2 years
Relapse-free survival
Will be estimated using the method of Kaplan and Meier.
Time frame: From the date of response to the date of documented relapses from CR or death from any cause, whichever occurs first, assessed up to 2 years
Overall survival
Will be estimated using the method of Kaplan and Meier.
Time frame: From treatment start till death or last follow-up, assessed up to 2 years
Proportion of patients proceeding to hematopoietic stem cell transplantation
Will also be estimated along with 95% credible interval.
Time frame: Up to 2 years
Incidence of adverse events
Time frame: Up to 30 days post-treatment
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