Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

Stopped early · Phase 3 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT04218084

Run by

Pfizer

People needed

236

Starts

2020-11-11

Expected to finish

2024-11-06

Last updated by the study team

2026-03-10

Who can join

Age: 2 and older, up to 14. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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
• 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
• 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

Where it is running

• Children's National Medical Center — Washington D.C., District of Columbia, United States
• University of Miami — Miami, Florida, United States
• Children's Healthcare of Atlanta: Hughes Spalding — Atlanta, Georgia, United States
• Boston Children's Hospital - Clinical Research Pharmacy — Boston, Massachusetts, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Medical University of South Carolina: Investigational Drug Services — Charleston, South Carolina, United States
• Medical University of South Carolina: Shawn Jenkins Women's and Children's Hospital — Charleston, South Carolina, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Texas Children's Hospital - Investigational Pharmacy — Houston, Texas, United States
• Texas Children's Hospital- Wallace Tower — Houston, Texas, United States
• Texas Children's Hospital — Houston, Texas, United States
• Ain Shams University Hospital- Clinical Research Center (MASRI) — Cairo, Abassia, Egypt
• Zagazig University Hospital — Zagazig, Alsharkia, Egypt
• Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University — Alexandria, Egypt
• Abu El Rich Hospital, Cairo University Hospital — Cairo, Egypt
• Komfo Anokye Teaching Hospital — Kumasi, Ashanti Region, Ghana
• Department of Child Health, University of Ghana Medical School, College of Health Sciences, Korle-Bu — Accra, Greater Accra Region, Ghana
• Azienda Ospedaliera Universitaria Meyer "A.O.U. Meyer" - SOC "Oncologia, Ematologia e TCSE" — Florence, Italy
• Azienda Ospedaliera Universitaria (A.O.U.) "Luigi Vanvitelli" — Naples, Italy
• Azienda Ospedaliera Universita' (AOU ) Padova — Padova, Italy
• Azienda Ospedaliera Universita' (AOU) Padova — Padova, Italy
• Kemri/Crdr,Siaya,Kemri Clinical Research Annex — Kisumu, Siaya County, Kenya
• KEMRI CRDR Clinical Research Annex — Nairobi, Kenya

Full record on ClinicalTrials.gov

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