This research study is designed to selectively deplete CD117-positive cells from participants with AML and MDS-EB.
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential anti-leukemia activity and to establish the minimum safe and biologically-effective dose of a single dose of MGTA-117 in relapsed/refractory (R/R) CD117+ AML participants and participants with MDS-EB. The study consists of escalating single-dose cohorts using a standard 3+3 design.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
MGTA-117 will be administered as an IV infusion
City of Hope
Duarte, California, United States
Sarah Cannon Research Institute at HealthONE
Denver, Colorado, United States
Moffitt Cancer Center
Tampa, Florida, United States
The University of Kansas Cancer Center
Westwood, Kansas, United States
Incidence rate of treatment emergent adverse events (TEAEs) leading to study drug discontinuation
Time frame: 21 days
Incidence rate of treatment emergent >= Grade 3 clinical laboratory abnormalities as assessed by CTCAE v5.0
Time frame: 21 days
Assess the clinically significant changes from baseline in vital signs, ECGs and laboratory parameters
Time frame: 21 days
Pharmacokinetics profile of MGTA-117
Investigate area under the curve (AUC)
Time frame: 21 days
Pharmacokinetics profile of MGTA-117
Investigate maximum plasma concentration (Cmax)
Time frame: 21 days
Pharmacokinetics profile of MGTA-117
Investigate time of maximum concentration (Tmax)
Time frame: 21 days
Pharmacokinetics profile of MGTA-117
Investigate the half-life (t1/2)
Time frame: 21 days
Pharmacokinetics profile of MGTA-117
Investigate the plasma concentration
Time frame: 21 days
To establish a minimum safe and biologically effective dose
Assess the CD117 receptor occupancy in circulating leukemic blasts
Time frame: 7 days
To establish a minimum safe and biologically effective dose
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University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Roswell Park Comprehensive Cancer Center
Buffalo, New York, United States
MD Anderson Cancer Center
Houston, Texas, United States
The incidence of qualifying protocol-defined dose-limiting toxicities
Time frame: 21 days