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.
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).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
240
Daily (QD), continuous dosing
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
Azacitidine will be given to study participants in Part D as intravenous infusion at a dose of 75 mg/m\^2
The Kirklin Clinic of UAB Hospital
Birmingham, Alabama, United States
RECRUITINGCity of Hope Comprehensive Cancer Center
Duarte, California, United States
ACTIVE_NOT_RECRUITINGUniversity of California Irvine
Irvine, California, United States
RECRUITINGUCSD Moores Cancer Center
La Jolla, California, United States
Frequency and severity of drug-related adverse events
Time frame: 4 years
Maximum tolerated dose (MTD) of tuspetinib
The MTD will be determined as the dose at which no more than 1 out of 6 study participants experiences a dose-limiting toxicity (DLT). Alternatively, the safety of a clinically effective dose below the MTD will be established if the MTD is not reached in study participants.
Time frame: 4 years
Maximum plasma concentration (Cmax)
Maximum plasma concentration (Cmax) will be summarized by cohort using descriptive statistics including number of study participants, mean, standard deviation, minimum, median, maximum, geometric mean, and coefficient of variation (CV) of the mean and geometric mean. Time-course of drug concentrations will be plotted as appropriate.
Time frame: Cycle 1 (at least 28 days)
Minimum plasma concentration (Cmin)
Minimum plasma concentration (Cmin) will be summarized by cohort using descriptive statistics including number of study participants, mean, standard deviation, minimum, median, maximum, geometric mean, and coefficient of variation (CV) of the mean and geometric mean. Time-course of drug concentrations will be plotted as appropriate.
Time frame: Cycle 1 (at least 28 days)
Area under the plasma concentration-time curve (AUC)
Area under the plasma concentration-time curve (AUC) for various timepoints will be summarized by cohort using descriptive statistics including number of study participants, mean, standard deviation, minimum, median, maximum, geometric mean, and coefficient of variation (CV) of the mean and geometric mean. Time-course of drug concentrations will be plotted as appropriate.
Time frame: Cycle 1 (at least 28 days)
Time to maximum concentration (Tmax)
Time to maximum concentration (Tmax) will be summarized by cohort using descriptive statistics including number of study participants, mean, standard deviation, minimum, median, maximum, geometric mean, and coefficient of variation (CV) of the mean and geometric mean. Time-course of drug concentrations will be plotted as appropriate.
Time frame: Cycle 1 (at least 28 days)
Terminal half-life (t1/2)
Terminal half-life (t1/2) will be summarized by cohort using descriptive statistics including number of study participants, mean, standard deviation, minimum, median, maximum, geometric mean, and coefficient of variation (CV) of the mean and geometric mean. Time-course of drug concentrations will be plotted as appropriate.
Time frame: Cycle 1 (at least 28 days)
Volume of distribution
Volume of distribution at various timepoints will be summarized by cohort using descriptive statistics including number of study participants, mean, standard deviation, minimum, median, maximum, geometric mean, and coefficient of variation (CV) of the mean and geometric mean. Time-course of drug concentrations will be plotted as appropriate.
Time frame: Cycle 1 (at least 28 days)
Plasma clearance (CL)
Plasma clearance (CL) will be summarized by cohort using descriptive statistics including number of study participants, mean, standard deviation, minimum, median, maximum, geometric mean, and coefficient of variation (CV) of the mean and geometric mean. Time-course of drug concentrations will be plotted as appropriate.
Time frame: Cycle 1 (at least 28 days)
Recommended Phase 2 dose (RP2D) of tuspetinib
The RP2D will be based on safety, efficacy, pharmacokinetic (PK), and pharmacodynamic (PD) considerations.
Time frame: 4 years
Complete remission (CR)
Complete remission (CR) will be summarized using descriptive statistics.
Time frame: 4 years
Complete remission with partial hematologic recovery (CRh)
Complete remission with partial hematologic recovery (CRh) will be summarized using descriptive statistics.
Time frame: 4 years
Complete remission with incomplete platelet recovery (CRp)
Complete remission with incomplete platelet recovery (CRp) will be summarized using descriptive statistics.
Time frame: 4 years
Complete remission with incomplete hematologic recovery (CRi)
Complete remission with incomplete hematologic recovery (CRi) will be summarized using descriptive statistics.
Time frame: 4 years
Partial remission (PR)
Partial remission (PR) will be summarized using descriptive statistics.
Time frame: 4 years
Overall response rate
Overall response rate will be summarized using descriptive statistics.
Time frame: 4 years
Duration of response
Survival curves and median for time-to-event variables will be estimated using the Kaplan-Meier method and will be reported along with corresponding 95% confidence intervals.
Time frame: 4 years
Disease-free survival (DFS)
Survival curves and median for time-to-event variables will be estimated using the Kaplan-Meier method and will be reported along with corresponding 95% confidence intervals.
Time frame: 4 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
USC/Norris Comprehensive Cancer Center
Los Angeles, California, United States
RECRUITINGStanford Cancer Center
Palo Alto, California, United States
RECRUITINGUniversity of California, Davis
Sacramento, California, United States
RECRUITINGYale University
New Haven, Connecticut, United States
RECRUITINGUniversity of Miami - Miller School of Medicine
Miami, Florida, United States
RECRUITINGEmory University
Atlanta, Georgia, United States
ACTIVE_NOT_RECRUITING...and 24 more locations
Overall survival (OS)
Survival curves and median for time-to-event variables will be estimated using the Kaplan-Meier method and will be reported along with corresponding 95% confidence intervals.
Time frame: 4 years
Event-free survival (EFS)
Survival curves and median for time-to-event variables will be estimated using the Kaplan-Meier method and will be reported along with corresponding 95% confidence intervals.
Time frame: 4 years