This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.
| Stacy Tsukayama | |
| 650.822.8729 | |
| [email protected] |
| Margaret Tonda, PharmD | |
| (650) 741-7761 | |
| [email protected] |
17 United States sites
5 Nigeria sites
4 Egypt sites
4 United Kingdom sites
3 Italy sites
3 Kenya sites
2 Ghana sites
2 Oman sites
2 Saudi Arabia sites
1 France site
2 to 14 Years
SCA
Phase 3
Interventional
2 Years - 14 Years
All
Drug
Unknown
Male or female participants with Sickle Cell Anemia (SCA)
TCD time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow ≥ 170 to < 200cm/sec during the Screening Period
Hb ≥ 5.5 and ≤ 10.5 g/dL during screening
For participants taking HU, the dose of HU (mg/kg) must be stable for at least 90 days prior to signing the informed consent form (ICF) and/or assent form, and with no anticipated need for dose adjustments (other than weight based) or for initiation of HU for non-chronic use during the study, in the opinion of the Investigator
Written informed parental/guardian consent and participant assent (where applicable) has been obtained per IRB/EC policy and requirements, consistent with ICH guidelines.
Body weight < 10kg at the screening visit Hospitalization for VOC or acute chest syndrome (ACS) within the 14 days prior to execution of informed consent/assent (see Section 7.2.5.1 for the definition of VOC). More than 10 VOCs within the past 12 months that required hospitalization, emergency room, or clinic visit Stroke resulting in focal neurological deficit; previous silent infarcts are permitted. Known history or findings suggestive of significant cerebral vasculopathy (eg, moyamoya or significant vasculopathy) History of seizure disorder Has been treated with erythropoietin or other hematopoietic growth factors within 28 days of signing informed consent/assent or if, in the opinion of the Investigator, there is an anticipated need for such agents during the study RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion) or has received an RBC transfusion or exchange transfusion for any reason within 90 days of signing the informed consent/assent