Safety, Tolerability and Pharmacokinetics of Milademetan Alone and With 5-Azacitidine (AZA) in Acute Myelogenous Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS) | Aplastic Anemia & MDS International Foundation
Safety, Tolerability and Pharmacokinetics of Milademetan Alone and With 5-Azacitidine (AZA) in Acute Myelogenous Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS)

Clinical Trial: NCT02319369

For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

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 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 AML
  2. newly diagnosed AML unfit for intensive chemotherapy
  3. high-risk 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.

Status: 
Recruiting
Study Date: 
Thu, 12/18/2014 to Thu, 07/01/2021
Bone Marrow Disease(s): 
  • acute myeloid leukemia (AML)
  • myelodysplastic syndromes (MDS)
Associated Drug(s): 
Intervention: 
-Drug: Milademetan Milademetan will be administered as a single oral capsule or as a combination of multiple oral capsules containing 5 mg, 20 mg, 80 mg, and/or 200 mg milademetan Other Name: Oral MDM2 Inhibitor -Drug: AZA AZA will be administered at 75 mg/m^2 subcutaneously or intravenously Other Name: 5-Azacitidine