Open-Label Extension of Voxelotor
- Conditions
- Sickle Cell DiseaseHematology, Sickle Cell, Disorder
- Registration Number
- LBCTR2020063513
- Lead Sponsor
- Global Blood Therapeutics Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- All
- Target Recruitment
- 21
1. Male or female participant with SCD, aged = 4 years, who participated and received study drug in a GBT-sponsored voxelotor pediatric clinical study
Note: Participants 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.
2. Female participants of childbearing potential are required to have a negative urine pregnancy test before dosing on Day 1.
Note: Female participants who become childbearing during the study must be willing to have a negative urine pregnancy test to remain in the study.
3. If sexually active, female participants of childbearing potential must use highly effective methods of contraception until 30 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 30 days after the last dose of study drug.
4. Participant has provided written assent (both the consent of the participant’s legal representative or legal guardian and the participant’s assent
[where applicable] must be obtained)
5. Subjects with abnormal TCD who have not completed Study GBT440-032 can participate in OLE study
1. Female participant who is breastfeeding or pregnant
2. Participant withdrew consent from a GBT-sponsored voxelotor pediatric clinical study
3. Participant was lost to follow-up from a GBT-sponsored voxelotor pediatric clinical study
4. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the investigator, may confound safety interpretation,
interfere with compliance, or preclude informed consent
5. Based on the most recent Oxbryta® US label (December 2021), co-administration with both moderate and strong CYP3A4 inducers should be avoided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ame: Treatment Emergent Adverse Events and Serious Adverse Events;Timepoints: Throughout entire study;Measure: N/A;Name: Sickle Cell Disease-Related Complications;Timepoints: Throughout entire study;Measure: Frequency of SCD-related complications
- Secondary Outcome Measures
Name Time Method ame: N/A;Timepoints: N/A;Measure: N/A