Study of Milademetan in Japanese Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Daiichi Sankyo

Status and phase: Completed; Phase 1

Conditions: Acute Myeloid Leukemia

Treatments: Drug: Milademetan

Study type: Interventional

Funder types: Industry

Identifiers

NCT03671564

184054 (Registry Identifier)

DS3032-A-J104

Details and patient eligibility

About

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).

Enrollment: 14 patients

Sex: All

Ages: 20+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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14 participants in 3 patient groups

Milademetan (90 mg/Day)

Experimental group

Description:

Participants who received milademetan 90 mg daily (QD) Day 1 - 14 followed by 14-day rest in a 28-day cycle.

Treatment:

Drug: Milademetan

Milademetan (120 mg/Day)

Experimental group

Description:

Participants who received milademetan 120 mg daily (QD) Day 1 - 14 followed by 14-day rest in a 28-day cycle.

Treatment:

Drug: Milademetan

Milademetan (160 mg/Day)

Experimental group

Description:

Participants who received milademetan 160 mg daily (QD) Day 1 - 14 followed by 14-day rest in a 28-day cycle.

Treatment:

Drug: Milademetan