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)
Aptose Biosciences Inc.
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.
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 pa…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria for Parts A/B/C: * Study participant is defined as having morphologically documented primary or secondary AML, MDS-IB2 (≥ 10% bone marrow blasts), or CMML by the World Health Organization (WHO) criteria (2016), and fulfills one of the following: 1. Refractory to at least 1 cycle of prior therapy 2. Relapsed after achieving remission with a prior therapy * Study participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2. * Study participant's interval from prior treatment to time of study drug administration is at least 2 weeks for cytotoxic age…
Interventions
- DrugTuspetinib
Daily (QD), continuous dosing
- DrugVenetoclax 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
- DrugAzacitidine for Intravenous Infusion
Azacitidine will be given to study participants in Part D as intravenous infusion at a dose of 75 mg/m\^2
Locations (34)
- The Kirklin Clinic of UAB HospitalBirmingham, Alabama
- City of Hope Comprehensive Cancer CenterDuarte, California
- University of California IrvineIrvine, California
- UCSD Moores Cancer CenterLa Jolla, California
- USC/Norris Comprehensive Cancer CenterLos Angeles, California
- Stanford Cancer CenterPalo Alto, California