An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
This is an open label, single arm study which will enroll up to 16 subjects with SCD who previously participated in the GBT440-001 study.
Dosing of study drug will be for 2 to 6 months, depending on subject's dose assignment in the last administration of study drug in GBT440-001.
The primary objective of the study is 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.
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
|Official Title:||An Open Label Single Arm Extension Study to Further Evaluate the Safety, Tolerability and Treatment Response of GBT440 in Patients With Sickle Cell Disease Who Participated in the Phase 1 Study GBT440-001|
- The Incidence of Treatment-Emergent Adverse Events during dosing of GBT440 for up to 6 months. [ Time Frame: 2 - 6 months ]The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
- To assess the efficacy of GBT440 as measured by improvements in anemia [ Time Frame: 2 - 6 months ]Measurement of haemoglobin
- To observed pharmacokinetics in plasma and whole blood. [ Time Frame: 2 - 6 months ]Measure maximum plasma concentration (Cmax)
- To characterize the effect of GBT440 on hemolysis. [ Time Frame: 2 - 6 months ]Measurement of hemolysis include unconjugated bilirubin.
|Study Start Date:||August 2016|
|Estimated Study Completion Date:||August 2017|
|Estimated Primary Completion Date:||May 2017 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT03041909
|The BRC Research Facility, Floor 15 The Tower Wing|
|London, United Kingdom, SE1 9RT|
|Study Director:||Josh Lehrer-Graiwer, MD||Global Blood Therapeutics, Inc.|
|Principal Investigator:||Timothy Mant, FRCP, FFPM||Quintiles, Inc.|