Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
Reason given: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment.
Key facts
- Where
- Atlanta, Boston, Charleston, Houston, Jackson, Miami, Philadelphia, and Washington D.C., United StatesSee the 13 sites
- Phase
- Phase 3
Where in United States
- Children's Healthcare of Atlanta: Hughes SpaldingAtlanta, United StatesNo longer taking part
- Boston Children's HospitalBoston, United StatesNo longer taking part
- Boston Children's Hospital - Clinical Research PharmacyBoston, United StatesNo longer taking part
- Medical University of South CarolinaCharleston, United StatesNo longer taking part
- Medical University of South Carolina- Investigational Drug ServicesCharleston, United StatesNo longer taking part
- Medical University of South Carolina: Shawn Jenkins Women's and Children's HospitalCharleston, United StatesNo longer taking part
- Texas Children's HospitalHouston, United StatesNo longer taking part
- Texas Children's Hospital - Investigational PharmacyHouston, United StatesNo longer taking part
- Texas Children's Hospital - Wallace TowerHouston, United StatesNo longer taking part
- University of Mississippi Medical CenterJackson, United StatesNo longer taking part
- University of MiamiMiami, United StatesNo longer taking part
- The Children's Hospital Of PhiladelphiaPhiladelphia, United StatesNo longer taking part
- Children's National Medical CenterWashington D.C., United StatesNo longer taking part
Trial details
- Who runs it
- Pfizer
- Trial code
- NCT04218084
- Record updated
- 10 March 2026
- Original description
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.Read full original descriptionShow less
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease (HOPE Kids 2)
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.
Inclusion: Male or female participants with Sickle Cell Anemia (SCA) HbSS, HbSβ0 thalassemia genotype; 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.
Exclusion: Hospitalization for VOC or acute chest syndrome (ACS) within the 14 days prior to execution of informed consent/assent; Stroke resulting in focal neurological deficit; previous silent infarcts are permitted; 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; History of seizure disorder; Body weight < 10kg at the screening visit; More than 10 VOCs within the past 12 months that required hospitalization, emergency room, or clinic visit; Known history or findings suggestive of significant cerebral vasculopathy.