Brief Summary
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
Brief Title
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Detailed Description
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:
1. Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures.
2. Frequency of sickle cell disease (SCD)-related complications.
3. 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).
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:
1. Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures.
2. Frequency of sickle cell disease (SCD)-related complications.
3. 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).
Categories
Completion Date
Completion Date Type
Actual
Conditions
Sickle Cell Disease
Eligibility Criteria
Inclusion Criteria:
* Male or female study participants with SCD who participated and received study treatment in Study GBT440-031.
Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.
* Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
* Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug.
* Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).
Exclusion Criteria:
* Female who is breast-feeding or pregnant.
* Participant withdrew consent from Study GBT440-031.
* Participant was lost to follow-up from Study GBT440-031.
* Participant requiring chronic dialysis.
* Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.
* Male or female study participants with SCD who participated and received study treatment in Study GBT440-031.
Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.
* Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
* Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug.
* Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).
Exclusion Criteria:
* Female who is breast-feeding or pregnant.
* Participant withdrew consent from Study GBT440-031.
* Participant was lost to follow-up from Study GBT440-031.
* Participant requiring chronic dialysis.
* Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.
Inclusion Criteria
Inclusion Criteria:
* Male or female study participants with SCD who participated and received study treatment in Study GBT440-031.
Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.
* Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
* Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug.
* Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).
* Male or female study participants with SCD who participated and received study treatment in Study GBT440-031.
Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.
* Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
* Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug.
* Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).
Gender
All
Gender Based
false
Keywords
Open Label Extension
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
65 Years
Minimum Age
12 Years
NCT Id
NCT03573882
Org Class
Industry
Org Full Name
Pfizer
Org Study Id
GBT440-034
Overall Status
Terminated
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Actual
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
Primary Outcomes
Outcome Description
Safety based on Adverse Events
Outcome Measure
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Outcome Time Frame
Five Years
Outcome Description
Frequency of SCD-related complications with long-term dosing with voxelotor.
Outcome Measure
Frequency of sickle cell-related complications
Outcome Time Frame
Five Years
Secondary Ids
Secondary Id
C5341022
Secondary Outcomes
Outcome Description
Measured by Hemoglobin, Bilirubin, and Reticulocyte counts
Outcome Time Frame
Five Years
Outcome Measure
Response in Hemolytic Anemia
See Also Links
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Locked Fields
Render the field
Maximum Age Number (converted to Years and rounded down)
65
Minimum Age Number (converted to Years and rounded down)
12
Investigators
Investigator Type
Principal Investigator
Investigator Name
Caterina Minniti
Investigator Email
caterina.minniti@einsteinmed.org