🏛️ Verified NIH Record • Updated 2026-04-13

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 (NCT04140487)

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

🟢 Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic Sponsor: M.D. Anderson Cancer Center

🩺 Plain-English Summary (8th-Grade Level)

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.

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (11 items) and avoid the listed exclusions (17 items).

Location: M D Anderson Cancer Center — Houston, Texas

Age range: 18 Years

🧪 Interventions in This Study

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

Usually NOT eligible if any of the following apply:

  • Exclusion Criteria:
  • Prior therapies:
  • Phase I cohort: No restriction based on prior therapies
  • Phase II cohort A: Patients with prior therapy for AML are not eligible. 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 B: No restriction on number of prior therapies
  • Patients suitable for and willing to receive intensive induction chemotherapy (for Phase II cohort A only)
  • Congenital long QT syndrome or corrected QT interval by Fridericia (QTcF) \> 450 msec. Repeat electrocardiograms (EKGs) after correction of electrolytes or discontinuation of QT prolonging medications are allowed to meet entry criteria. In cases where QTcF \> 450 msec is considered to be falsely increased due to inaccurate automated reading and not clinically significant (e.g. due to bundle branch block), patients are still eligible if cardiologist reviews and documents that QTcF is ≤ 450 msec when manually measured
  • Active serious infection not controlled by oral or intravenous antibiotics (e.g. persistent fever or lack of improvement despite antimicrobial treatment)
  • Active grade III-V cardiac failure as defined by the New York Heart Association criteria
  • Active central nervous system leukemia
  • Known human immunodeficiency virus (HIV) seropositive
  • Known hepatitis B surface antigen seropositive or known or suspected active hepatitis C infection

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (1 Sites)

  • M D Anderson Cancer Center — Houston, Texas

📞 Contact the Study Coordinator

Phone: (713) 563-4485

Email: nshort@mdanderson.org

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Recurrent Acute Myeloid Leukemia

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💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Contact the study coordinator using the phone/email above.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗