An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
|ClinicalTrials.gov Identifier: NCT03041909|
Recruitment Status : Completed
First Posted : February 3, 2017
Last Update Posted : February 15, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: GBT440||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||August 2016|
|Actual Primary Completion Date :||June 2017|
|Actual Study Completion Date :||August 2017|
Experimental: Single Arm
Single Arm / open label
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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.|