TAK-243 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Refractory Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
Last updated on July 2021Recruitment
- Recruitment Status
- Not yet recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Myelodysplastic Syndrome
- Recurrent Acute Myeloid Leukemia
- Refractory Acute Myeloid Leukemia
- Refractory Chronic Myelomonocytic Leukemia
- Refractory High Risk Myelodysplastic Syndrome
- Type
- Interventional
- Phase
- Phase 1
- Design
- Allocation: Non-RandomizedIntervention Model: Sequential AssignmentMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 125 years
- Gender
- Both males and females
Description
PRIMARY OBJECTIVES: I. To establish the recommended phase 2 dose (RP2D) of ubiquitin-activating enzyme (UAE) inhibitor TAK-243 (MLN7243 [TAK-243]) administered intravenously in a twice-weekly schedule in patients with acute myeloid leukemia (AML) or patients with myelodysplastic syndromes (MDS) or c...
PRIMARY OBJECTIVES: I. To establish the recommended phase 2 dose (RP2D) of ubiquitin-activating enzyme (UAE) inhibitor TAK-243 (MLN7243 [TAK-243]) administered intravenously in a twice-weekly schedule in patients with acute myeloid leukemia (AML) or patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) refractory to hypomethylating agents (HMAs). SECONDARY OBJECTIVES: I. To assess the maximum tolerated dose (MTD) evaluated on the first cycle (Day 1 to 21) of MLN7243 (TAK-243), its safety profile, and dose limiting toxicities (DLT). II. To investigate preliminary anti-leukemic activity of MLN7243 (TAK-243) monotherapy in patients with AML, MDS and CMML. III. To relate responses to the molecular/cytogenetic abnormalities in the malignant cells or to pharmacokinetic (PK)/pharmacodynamic (PD) findings. IV. To describe the PK of MLN7243 (TAK-243). V. To describe the PD profile of MLN7243 (TAK-243) in the study population. VI. To establish the RP2D of MLN7243 (TAK-243) administered intravenously in a once-weekly schedule in patients with AML or patients with MDS or CMML refractory to HMAs. OUTLINE: This is a dose escalation, followed by a dose expansion, study. Patients are assigned to 1 of 2 groups. GROUP I: Patients receive UAE inhibitor TAK-243 intravenously (IV) over 10 minutes on days 1, 4, 8, and 11. Treatment repeats every 21 days for up to 12 months in the absence of disease progression or unacceptable toxicity. GROUP II: Patients receive UAE inhibitor TAK-243 IV over 10 minutes on days 1, 8, and 15. Treatment repeats every 28 days for up to 12 months in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days.
Tracking Information
- NCT #
- NCT03816319
- Collaborators
- Not Provided
- Investigators
- Principal Investigator: Dawn C Maze University Health Network Princess Margaret Cancer Center LAO