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Open, not recruiting

AML16: Phase 2 Trial of Epigenetic Priming in Newly Diagnosed Acute Myeloid Leukemia

About this study

Acute myelogenous leukemia (AML) is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone marrow. They may also invade body organs including the brain, testes, ovaries, or skin. These cancerous AML cells can sometimes form a solid tumor called a chloroma.

This study will compare the good and bad effects of giving either azacitidine OR decitabine before the usual chemotherapy regimen for childhood AML. Azacitidine and decitabine both belong to a class of drugs called "DNA methyl-transferase inhibitors" or DMTi. DMTi’s are known to alter the DNA within the genes of leukemia cells, possibly making them more sensitive to chemotherapy. Giving a DMTi before the usual chemotherapy is called "epigenetic priming" because the intent is to change the genetics of the leukemia cell by “priming” it to be more sensitive to the chemotherapy that will follow.

The U.S. Food and Drug Administration (FDA) has approved azacitidine and decitabine to treat adults with myelodysplastic syndrome. The FDA has not approved these drugs for treating children with leukemia.

Eligibility overview

  • Diagnosis of 1 of the following:
    • Acute myeloid leukemia (AML)
    • 5% to 20% marrow myeloblasts and evidence of a clonal de novo AML genetic abnormality
    • Myeloid sarcoma
    • High grade myelodysplastic syndrome (MDS) with greater than 5% blasts
    • Treatment-related myeloid neoplasms, including AML and MDS
  • 28 days to 21 years old
  • No prior therapy, except for one dose of intrathecal therapy and the use of hydroxyurea or low-dose cytarabine
  • Not pregnant

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.


Full title:

Phase 2 Trial of Epigenetic Priming in Patients with Newly Diagnosed Acute Myeloid Leukemia 

Study goal:

The main purpose of this study is to find out if adding either azacitidine or decitabine is better, the same, or worse than the usual chemotherapy for AML. Researchers also want to know if azacitidine or decitabine is safe, and if one of the drugs is better than the other.


28 days to 21 years old

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.


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