Phase 1
Completed N=14
Study of Milademetan in Japanese Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)
Source: ClinicalTrials.gov NCT03671564 ↗Enrolled (actual)
14
Serious AEs
28.6%
Results posted
Nov 2023
Primary outcomePrimary: Number of Participants With Dose Limiting Toxicities (DLTs) Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia — 0; 0; 0 Participants
Summary
This is a Phase 1, multicenter, open-label study to evaluate safety, tolerability and pharmacokinetics of milademetan in Japanese patients with relapsed or refractory acute myeloid leukemia. The milademetan initial dose will be Level 1: 90 mg. No increase in the milademetan dose will be made in the same participant. Dose-limiting toxicity associated with milademetan occurring at each level will be assessed, and the maximum tolerated dose (MTD) will be decided using a modified continuous reassessment method (mCRM).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Dose Limiting Toxicities (DLTs) Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
0; 0; 0 | — |
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
4; 5; 4 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of Milademetan Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
653; 1010; 1380; 1090; 1680; 2710 | — |
| SECONDARY Time to Reach Maximum Plasma Concentration (Tmax) of Milademetan Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
4.45; 3.13; 2.54; 3.02; 2.98; 3.03 | — |
| SECONDARY Area Under the Plasma Concentration-Time Curve (AUC) of Milademetan Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
3170; 4940; 6560; 6220; 10000; 15300 | — |
| SECONDARY Terminal Elimination Half-Life (T1/2) of Milademetan Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
15.9; 14.1; 14.6 | — |
| SECONDARY Trough Plasma Concentration (Ctrough) of Milademetan Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
422; 635; 769 | — |
| SECONDARY Number of Participants With Best Response Following Administration of Milademetan in Participants With Relapsed or Refractory Acute Myeloid Leukemia |
0; 0; 0; 0; 0; 0 | — |
Eligibility Criteria
Inclusion Criteria
- Relapsed or refractory AML
- AML for which no standard treatment is available
- Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 to 2
Exclusion Criteria
- Acute Promyelocytic Leukemia
- Chronic myelogenous leukemia in blast crisis (BCR-ABL fusion gene positive)
- Presence of central nervous system involvement of leukemia or a history of primary central nervous system leukemia
Data sourced from ClinicalTrials.gov (NCT03671564). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.