This is a dose escalation study that will be conducted in healthy volunteers. Multiple cohorts of volunteers will receive ascending oral doses of AG-348 to evaluate the safety and tolerability of a single oral dose of AG-348, the pharmacokinetics (PK) of a single dose of AG-348, and the pharmacodynamic profile of AG-348 with specific biomarkers assessed in the blood.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
48
Unnamed facility
Baltimore, Maryland, United States
Safety and tolerability
Incidence of adverse events and descriptive statistics for safety laboratory parameters, physical exam findings, vital signs and ECGs.
Time frame: 11 days
Pharmacokinetics of AG-348
Descriptive statistics will be used to summarize PK parameters of AG-348 for each dose group and, where appropriate, for the entire population. Standard non-compartmental PK parameters will be calculated from individual plasma concentration data.
Time frame: 4 days
Pharmacodynamic (PD) relationship of AG-348 and metabolic biomarkers
The potential relationship between AG-348 and metabolic biomarkers will be explored with descriptive and graphical methods. Details on the evaluation of exploratory analyses of pyruvate kinase (PKR) activity and other potential PD biomarkers will be described.
Time frame: 4 days
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