Pediatric Open-Label Extension of Voxelotor
Phase 3
- Conditions
- Haematological DisordersSickle Cell DiseasePaediatrics
- Registration Number
- PACTR202209867711506
- Lead Sponsor
- Global Blood Therapeutics inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- All
- Target Recruitment
- 300
Inclusion Criteria
1. Male or female participant with SCD, up to 18 years, who participated and received study drug in a GBT-sponsored voxelotor pediatric clinical study
2. 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)
Exclusion Criteria
1.Female participant who is breastfeeding or pregnant
2. Participant withdrew consent from a GBT-sponsored voxelotor pediatric clinical study
3. Known hypersensitivity to voxelotor or any other components of the study drug
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. TEAEs and SAEs [ Time Frame: Throughout entire study ]<br> Treatment Emergent Adverse Events and Serious Adverse Events<br>2. Sickle Cell Disease-Related Complications [ Time Frame: Throughout entire study ]<br> Frequency of SCD-related complications
- Secondary Outcome Measures
Name Time Method ot Provided