This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088). Dosing of study drug was 2 to 6 months, depending on subject's dose assignment in the last administration of study drug in GBT440-001 (NCT02285088). The primary objective of the study was to evaluate the safety and tolerability of up to a total of 6 months dosing of subjects with SCD who participated in the GBT440-001 study (NCT02285088).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
5
Oral drug
The BRC Research Facility, Floor 15 The Tower Wing
London, United Kingdom
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Time frame: 2 - 6 months
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
Data presented are hemoglobin value collected at specific time points.
Time frame: 2 - 6 months
To Observed Pharmacokinetics in Plasma and Whole Blood.
Measure maximum plasma concentration (Cmax)
Time frame: 2 - 6 months
To Characterize the Effect of GBT440 on Hemolysis.
Data presented for unconjugated bilirubin at specific time point.
Time frame: 2 - 6 months
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