NCT03816319

TAK-243 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndromes With Increased Blasts

A Phase 1 Study of TAK-243 for Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndromes With Increased Blasts

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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) profile of TAK-243. IV. To describe the pharmacodynamic (PD) effects of TAK-243. EXPLORATORY OBJECTIVE: I. To investigate associations between clinical responses and molecular/cytogenetic abnormalities in the leukemia cells or to the PK profile or PD effects of TAK-243. OUTLINE: This is a dose-escalation study. Patients receive TAK-243 intravenously (IV) over 30 minutes on days 1, 4, 8, and 11 of each cycle. Cycles repeats every 21 days for up to 12 months in the absence of disease progression or unacceptable toxicity. Patients undergo urine sample collection, bone marrow aspiration and bone marrow biopsy, multigated acquisition scan (MUGA) and echocardiogram (ECHO) during screening and on study, and blood sample collection throughout the trial. After completion of study treatment, patients are followed up at 30 days.

Conditions

• Myelodysplastic Syndrome With Excess Blasts
• Recurrent Acute Myeloid Leukemia
• Recurrent Myelodysplastic Syndrome
• Recurrent Myelodysplastic Syndrome/Acute Myeloid Leukemia
• Refractory Acute Myeloid Leukemia
• Refractory Myelodysplastic Syndrome
• Refractory Myelodysplastic Syndrome/Acute Myeloid Leukemia

Interventions

Type	Name	Description
PROCEDURE	Biospecimen Collection	Undergo urine and blood sample collection
PROCEDURE	Bone Marrow Aspiration	Undergo bone marrow aspiration
PROCEDURE	Bone Marrow Biopsy	Undergo bone marrow biopsy
PROCEDURE	Echocardiography Test	Undergo ECHO
PROCEDURE	Multigated Acquisition Scan	Undergo MUGA
DRUG	UAE Inhibitor TAK-243	Given IV

Timeline

• Start date: 2025-03-12
• Primary completion: 2026-10-24
• Completion: 2026-10-24
• First posted: 2019-01-25
• Last updated: 2026-04-13

Locations

5 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study