Last updated on February 2020

Safety Tolerability and Pharmacokinetics of Milademetan Alone and With 5-Azacitidine (AZA) in Acute Myelogenous Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS)


Brief description of study

This study will take place in parts:

  • Dose Escalation (Part 1): Participants receive milademetan alone with different dose schedules
  • Dose Escalation (Part 1A): Participants receive milademetan in combination with 5-azacytidine (AZA), with different dose schedules

The recommended dose for Part 2 will be selected.

  • Dose Expansion (Part 2): After Part 1A, participants will receive the recommended Part 2 dose schedule. There will be three groups - those with:
    1. refractory or relapsed acute myelogenous leukemia (AML)
    2. newly diagnosed AML unfit for intensive chemotherapy
    3. high-risk myelodysplastic syndrome (MDS)
  • End-of-Study Follow-Up: Safety information will be collected until 30 days after the last treatment. This is the end of the study.

The recommended dose for the next study will be selected.

Detailed Study Description

The primary analysis will occur after all participants have either discontinued the study or completed at least 6 months of treatment. After the primary analysis, the main study will be closed. Participants who are still on study at least 6 months after enrollment of the last participant in the study may be eligible to continue receiving study drug in a separate extension phase of the protocol

Clinical Study Identifier: NCT02319369

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