An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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.|