Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031 (034OLE)
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|ClinicalTrials.gov Identifier: NCT03573882|
Recruitment Status : Active, not recruiting
First Posted : June 29, 2018
Last Update Posted : March 17, 2020
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Voxelotor||Phase 3|
Expanded Access : An investigational treatment associated with this study has been approved for sale to the public. More info ...
This open label extension (OLE), multi-center study will be conducted at approximately 100 clinical sites globally and will be available to eligible participants from study GBT440-031.
The study will enroll participants from GBT440-031 (approximately 435) under any of the following conditions:
- Participant has completed 72 weeks of treatment regardless of dose selection for GBT440-031
- Dose selection has occurred for GBT440-031 and participant is on non-selected dose on GBT440-031
- GBT440-031 study interim data analysis and/or study modifications have occurred
- GBT440-031 study has completed
The objective of this open-label extension (OLE) study is to assess the long-term safety and treatment effect of voxelotor in participants who have completed treatment in study GBT440-031, using the following parameters:
- Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures.
- Frequency of sickle cell disease (SCD)-related complications.
- Hemolytic anemia as measured by hematological laboratory parameters (e.g. hemoglobin, reticulocytes and unconjugated bilirubin).
All participants will receive daily voxelotor treatment.
Participants may receive study drug as long they continue to receive clinical benefit which outweighs risk as determined by the Investigator and/or until the participant has access to voxelotor from an alternative source (i.e., commercialization or through a managed access program).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||179 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials|
|Actual Study Start Date :||June 6, 2018|
|Estimated Primary Completion Date :||October 2024|
|Estimated Study Completion Date :||October 2024|
Participants will receive voxelotor (GBT440) at the highest dose (either 900 mg or 1500 mg) deemed safe by the Data Safety Monitoring Board (DSMB).
300mg or 500mg Tablet, Oral, With or Without Food
Other Name: GBT440
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Five Years ]Safety based on Adverse Events
- Frequency of sickle cell-related complications [ Time Frame: Five Years ]Frequency of SCD-related complications with long-term dosing with voxelotor.
- Response in Hemolytic Anemia [ Time Frame: Five Years ]Measured by Hemoglobin, Bilirubin, and Reticulocyte counts
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): NCT03573882
|Study Director:||Margaret Tonda, PharmD||Global Blood Therapeutics|