PLX51107 and Azacitidine in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

  • participants needed
  • sponsor
    M.D. Anderson Cancer Center
Updated on 8 February 2023
myeloid leukemia
myeloproliferative disorder
ejection fraction
blast cells
colony stimulating factor
cytotoxic agents
myelodysplastic/myeloproliferative neoplasm
brd4 inhibitor plx51107


This phase I trial studies the side effects and best dose of PLX51107 and how well it works with azacitidine in treating patients with acute myeloid leukemia or myelodysplastic syndrome. PLX51107 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. 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. Giving PLX51107 and azacitidine may work better than azacitidine alone in treating patients with acute myeloid leukemia or myelodysplastic syndrome.



I. To determine the minimum safe and biologically-effective dose of the combination of PLX51107 and azacitidine (AZA).


I. To determine overall response rate (ORR) rate including CR (complete remission) + CRp (complete remission with incomplete platelet recovery) + CRi (complete remission with incomplete count recovery) + CRh (complete remission with partial hematologic recovery), partial remission (PR) within 3 months of treatment initiation of PLX51107 and AZA combination in patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).

II. To investigate correlations of response to this combination with a pre-therapy, on-therapy, and progression 81-gene panel of gene mutations in AML.

III. To determine the duration of response (DOR), event-free survival (EFS), overall survival (OS), and number of patients bridged to stem cell transplant (SCT) and median duration to SCT from the initiation of the combination.

OUTLINE: This is a dose-escalation study of PLX51107.

Patients receive PLX51107 orally (PO) once daily (QD) on days 1-21 and azacitidine subcutaneously (SC) or intravenously (IV) over 15 minutes on days 8-14 and 22-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days, then every 6-12 months for 5 years.

Condition Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasm, Myeloproliferative Neoplasm
Treatment Azacitidine, BRD4 Inhibitor PLX51107
Clinical Study IdentifierNCT04022785
SponsorM.D. Anderson Cancer Center
Last Modified on8 February 2023

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