A Phase 1 Study of TAK-243 for Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndromes With Increased Blasts
National Cancer Institute (NCI)
Summary
This phase I trial studies the side effects and best dose of TAK-243 in treating patients with acute myeloid leukemia or myelodysplastic syndromes with increased blasts that has come back (relapsed) or that is not responding to treatment (refractory). TAK-243 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Description
PRIMARY OBJECTIVE: I. To establish the recommended phase 2 dose (RP2D) of UAE inhibitor TAK-243 (TAK-243) administered intravenously in a twice-weekly schedule in patients with relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). SECONDARY OBJECTIVES: I. To assess the maximum tolerated dose (MTD) evaluated on the first cycle (Day 1 to 21) of TAK-243, its safety profile, and dose limiting toxicities (DLT). II. To investigate preliminary anti-leukemic activity of TAK-243 monotherapy in patients with AML or MDS. III. To describe the pharmacokinetic (PK) profil…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Diagnosis of AML or MDS with increased blasts (MDS-IB) assessed by local laboratory review according to the 2022 World Health Organization (WHO) criteria for myeloid neoplasms. Both patients with MDS-IB1 (5-9% bone marrow blasts) and MDS-IB2 (10-19% bone marrow blasts) are eligible. * Patients must have relapsed or refractory disease after receiving at least one prior line of therapy * AML-specific inclusion criteria: Patients with relapsed or refractory AML with \>= 5% bone marrow blasts after receiving at least two courses of intensive induction chemotherapy (including…
Interventions
- ProcedureBiospecimen Collection
Undergo urine and blood sample collection
- ProcedureBone Marrow Aspiration
Undergo bone marrow aspiration
- ProcedureBone Marrow Biopsy
Undergo bone marrow biopsy
- ProcedureEchocardiography Test
Undergo ECHO
- ProcedureMultigated Acquisition Scan
Undergo MUGA
- DrugUAE Inhibitor TAK-243
Given IV
Locations (5)
- Moffitt Cancer CenterTampa, Florida
- Northwestern UniversityChicago, Illinois
- Ohio State University Comprehensive Cancer CenterColumbus, Ohio
- VCU Massey Comprehensive Cancer CenterRichmond, Virginia
- University Health Network-Princess Margaret HospitalToronto, Ontario