NCT02319369

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

A Phase 1 Study of Milademetan (DS 3032b), an Oral MDM2 Inhibitor, In Dose Escalation as a Single Agent and In Dose Escalation/Expansion In Combination With 5 Azacitidine In Subjects With Acute Myelogenous Leukemia (AML) or High Risk Myelodysplastic Syndrome (MDS)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 74 (actual)
• Sponsor: Daiichi Sankyo · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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 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

Conditions

• Acute Myelogenous Leukemia
• Myelodysplastic Syndrome

Interventions

Type	Name	Description
DRUG	Milademetan	Milademetan will be administered daily as oral capsules or as a combination of multiple oral capsules containing 5 mg, 20 mg, 80 mg, and/or 200 mg
DRUG	AZA	AZA will be administered at 75 mg/m\^2 subcutaneously or intravenously
DRUG	Milademetan	Milademetan will be administered daily as oral capsules or as a combination of multiple oral capsules containing 5, 20, 80, and/or 200 mg. An alternate combination of 30 mg, 80 mg, and/or 100 mg milademetan may be utilized.

Timeline

• Start date: 2014-11-25
• Primary completion: 2020-08-21
• Completion: 2020-08-21
• First posted: 2014-12-18
• Last updated: 2021-10-11
• Results posted: 2021-10-11

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study