This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.
This is a multicenter, open-label, Phase Ia/b dose escalation study of safety, pharmacodynamics, and pharmacokinetics of APTO-253 in ascending cohorts (3+3 design) to determine the MTD or recommended dose in patients with relapsed or refractory acute myelogenous leukemia (AML) or high-risk MDS patients. This is to be followed by a cohort expansion phase at the MTD or recommended dose.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
21
APTO-253 will be given in ascending doses starting at 20 mg/m2 until the maximum tolerated dose or recommended dose is reached.
University of Arizona Cancer Center
Tucson, Arizona, United States
UC San Diego Moores Cancer Center
La Jolla, California, United States
University of California, Irvine
Orange, California, United States
Emory University; Winship Cancer Institute
Atlanta, Georgia, United States
Ochsner Cancer Institute
New Orleans, Louisiana, United States
University of Michigan
Ann Arbor, Michigan, United States
St. Vincent Frontier Cancer Center
Billings, Montana, United States
University of Rochester; Wilmot Cancer Institute Clinical Trials Office
Rochester, New York, United States
University Hospital
Cleveland, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
...and 3 more locations
Incidence of treatment-emergent adverse events of APTO-253
To determine the safety and tolerability of APTO-253 by assessing treatment-related adverse events as assessed by CTCAE v4.0.
Time frame: Cycle 1 (28 days)
Maximum tolerated dose and dose limiting toxicities
To determine the maximum tolerated dose (MTD) and the dose limiting toxicities (DLT) of APTO-253 when given on days 1, 8, 15, and 22 of each 28-day cycle.
Time frame: Cycle 1 (28 days)
Establish recommended dose for future development of APTO-253
To establish the dose of APTO-253 recommended for future development of APTO-253 for patients with specific types of hematologic malignancies.
Time frame: Up to 7 months
Pharmacokinetic variables including maximum plasma concentration (Cmax)
Pharmacokinetic variables including maximum plasma concentration (Cmax)
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including minimum plasma concentration (Cmin)
Pharmacokinetic variables including minimum plasma concentration (Cmin)
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including Area Under the Curve (AUC)
Pharmacokinetic variables including Area Under the Curve (AUC)
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including volume of distribution
Pharmacokinetic variables including volume of distribution
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including clearance
Pharmacokinetic variables including clearance
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including serum half-life
Pharmacokinetic variables including serum half-life
Time frame: Cycle 1 (28 days)
Assess for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.
To observe patients for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.
Time frame: Average 2 Cycles (8 weeks)
Determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.
To determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.
Time frame: Average 2 Cycles (8 weeks)
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