This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
129
Administered orally with water as a single dose in the morning.
ICON Early Phase Services, LLC
San Antonio, Texas, United States
Harry Perkins Institute of Medical Research
Nedlands, Western Australia, Australia
Linear Clinical Research
Nedlands, Western Australia, Australia
Oxford Compounding
North Perth, Western Australia, Australia
Safety, as assessed by frequency and severity of adverse events (AEs)
AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Time frame: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in Heart Rate.
Number of participants with changes in heart rate (bpm) as compared to baseline.
Time frame: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in eGFR
Number of participants with changes in eGFR from baseline
Time frame: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in alanine aminotransferase (ALT)
Number of participants with changes in alanine aminotransferase (ALT)
Time frame: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in Blood pressure
Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Time frame: 119 days from screening Part A, 134 days from screening Part B
Plasma concentration
Time of Cmax
Time frame: 119 days from screening Part A
Plasma concentration
Cmax on D1-D15
Time frame: 134 days from screening Part B
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Determine whole blood concentration of GBT021601
Hemoximetry will be used to assess oxygen saturation in whole blood by generating oxygen equilibrium curves (OECs) which relate the extent of Hb-O2 saturation to the partial pressure of O2 (pO2) and measure the binding affinity of O2 to Hb.
Time frame: 119 days from screening Part A
Determine plasma concentration of GBT021601.
With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).
Time frame: 134 days from screening Part B
Safety, as assessed by changes in QTcF
Number of participants with changes in the QTcF interval from baseline
Time frame: 119 days from screening Part A, 134 days from screening Part B