NCT03850574

Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1/2 Open Label, Multicenter, Dose Escalation and Expansion Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of HM43239 in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 240 (estimated)
• Sponsor: Aptose Biosciences Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that is relapsed or refractory after at least one line of prior therapy, or in study participants with newly diagnosed AML. Tuspetinib will be administered as a single agent or in combination with other drugs (venetoclax or venetoclax plus azacitidine), as specified for each part of the study.

Detailed description

This is a Phase 1/2, open-label, multi-center study designed to assess the efficacy, safety, tolerability, and pharmacokinetics, including determining the recommended Phase 2 dose (RP2D) of tuspetinib (HM43239) in subjects with relapsed or treatment-refractory acute myeloid leukemia (AML). Part C: This portion of the study will evaluate tuspetinib (HM43239) as monotherapy in patients with relapsed or refractory (R/R) AML, focusing on safety, tolerability, pharmacokinetics, and preliminary efficacy (Aptivate). Part D: This portion of the study will evaluate the safety, tolerability, and PK parameters of tuspetinib (HM43239) in combination with venetoclax and azacitidine when administered to newly diagnosed AML patients who are ineligible for induction chemotherapy (Tuscany).

Conditions

• Leukemia, Myeloid, Acute
• Refractory AML
• Relapsed Adult AML
• Myelodysplastic Syndrome With Excess Blasts-2
• Chronic Myelomonocytic Leukemia

Interventions

Type	Name	Description
DRUG	Tuspetinib	Daily (QD), continuous dosing
DRUG	Venetoclax Oral Tablet	Venetoclax will be given to study participants in the Part C tuspetinib plus venetoclax combination treatment group either in 50 mg or 100 mg tablets
DRUG	Azacitidine for Intravenous Infusion	Azacitidine will be given to study participants in Part D as intravenous infusion at a dose of 75 mg/m\^2

Timeline

• Start date: 2019-03-11
• Primary completion: 2026-11-01
• Completion: 2027-04-01
• First posted: 2019-02-22
• Last updated: 2025-08-26

Locations

34 sites across 6 countries: United States, Australia, Germany, New Zealand, South Korea, Spain

Regulatory

• FDA-regulated drug study