Azacitidine, Venetoclax, and Gilteritinib in Newly Diagnosed and Relapsed or Refractory FLT3-Mutated AML.
Nicholas J Short, Naval Daver, Courtney D Dinardo...
https://pubmed.ncbi.nlm.nih.gov/38277619Actively Recruiting
Phase 1
Phase 2
Age: 18Years +
All Genders
ID04140487
A Phase I/II Study of Azacitidine, Venetoclax, and Gilteritinib for Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome With an Activating FLT3 Mutation
Led by M.D. Anderson Cancer Center · Updated on 2026-04-16
97
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the combination of azacitidine, venetoclax, and gilteritinib in patients with FLT3-mutation positive acute myeloid leukemia, chronic myelomonocytic leukemia, or high-risk myelodysplastic syndrome/myeloproliferative neoplasm that has returned or not responded to treatment. This phase I/II trial aims to find the best dose and evaluate how well the combination works compared to azacitidine and venetoclax alone. The study also explores genetic factors that may affect treatment response and survival. Participants receive azacitidine through injection under the skin or into a vein for 7 days, venetoclax orally daily for 28 days in the first cycle and for 21 days in later cycles, and gilteritinib orally daily for 28 days. Treatment cycles repeat every 28 days for up to 24 cycles, unless the disease progresses or side effects become unacceptable. The study has a dose-escalation phase to determine the maximum tolerated dose of gilteritinib, followed by a phase II treatment phase. During the study, patients are monitored through regular visits to assess response, side effects, and overall health. Researchers measure outcomes like complete remission rates, minimal residual disease, relapse-free survival, and overall survival over up to 3 years. After treatment ends, patients have follow-up visits at 30 days and every 6 months to continue assessing their condition and safety.
CONDITIONS
Brief 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
You may qualify if you...
- Adults aged 18 years or older
- Diagnosis of FLT3-mutated acute myeloid leukemia, chronic myelomonocytic leukemia, or intermediate-2 or high-risk myelodysplastic syndrome/myeloproliferative neoplasm
- Performance status of 3 or less on the ECOG scale
- Total serum bilirubin less than or equal to 2.5 times the upper limit of normal unless due to specific approved causes
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) less than or equal to 3 times the upper limit of normal unless due to underlying leukemia
- Creatinine clearance of at least 30 mL/min
- Ability to swallow medication
- Signed informed consent
You will not qualify if you...
- For newly diagnosed patients, prior therapy for acute myeloid leukemia (except certain allowed treatments) excludes participation
- Patients suitable for and willing to receive intensive induction chemotherapy (newly diagnosed group only)
- Congenital long QT syndrome or corrected QT interval greater than 450 msec unless reviewed and approved
- Active serious infection uncontrolled by antibiotics
- Active grade III-V cardiac failure
- Active central nervous system leukemia
- Known HIV infection
- Known or suspected active hepatitis B or C infection without undetectable viral load
- Use of strong CYP3A or p-glycoprotein inducers within 3 days before enrollment
- Treatment with investigational antileukemic or chemotherapy agents within 7 days before study entry unless recovered from side effects
- Pregnant or breastfeeding women; women of childbearing potential must have negative pregnancy test and use contraception; men must use contraception during and after the study period
AI-Screening
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 24 cycles of 28 days each
Participants receive azacitidine by subcutaneous or intravenous injection on days 1-7, venetoclax orally daily on days 1-28 of the first cycle and days 1-21 of subsequent cycles, and gilteritinib orally daily on days 1-28. Treatment cycles repeat every 28 days for up to 24 cycles unless there is disease progression or unacceptable toxicity.
Visits occur with each treatment cycle every 28 days
Follow-up
Duration - Initial follow-up at 30 days post-treatment, then every 6 months thereafter
After completing study treatment, participants are followed up to monitor health status and outcomes.
Follow-up visits at 30 days and every 6 months
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
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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