A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease

About the study

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.

Study point of contact

Jason Farthing
Mark Davis


17 United States sites

5 Nigeria sites

4 Egypt sites

4 Kenya sites

4 United Kingdom sites

3 Italy sites

2 Ghana sites

2 Oman sites

2 Saudi Arabia sites

1 France site


2 Years - 14 Years




Phase 3

Study type






participation requirements

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.

participation restrictions

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 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


  • Birmingham, Alabama, United States, University of Alabama at Birmingham, 35233 [Recruiting]
  • Washington, District of Columbia, United States, Children's National Medical Center, 20010 [Terminated]
  • Miami, Florida, United States, University of Miami Medical Center, 33136 [Recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta-Hughes Spalding, 30303 [Terminated]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta- Scottish Rite/ EMORY, 30342 [Terminated]
  • Chicago, Illinois, United States, Ann & Robert H. Lurie Children's Hospital of Chicago, 60611 [Withdrawn]
  • Boston, Massachusetts, United States, Boston Children's Hospital, 02111 [Recruiting]
  • Detroit, Michigan, United States, Wayne State University, 48202 [Withdrawn]
  • Minneapolis, Minnesota, United States, Children's Health Care d/b/a Children's Minnesota, 55404 [Withdrawn]
  • Jackson, Mississippi, United States, University of Mississippi Medical Center, 39216 [Recruiting]
  • Kansas City, Missouri, United States, Children's Mercy Hospitals & Clinics, 64108 [Withdrawn]
  • Saint Louis, Missouri, United States, Washington University School of Medicine, 63110 [Recruiting]
  • Philadelphia, Pennsylvania, United States, Children's Hospital of Philadelphia, 19104 [Not yet recruiting]
  • Pittsburgh, Pennsylvania, United States, Children's Hospital of Pittsburgh of UPMC, 15224 [Not yet recruiting]
  • Charleston, South Carolina, United States, Medical University of South Carolina, 29425 [Recruiting]
  • Memphis, Tennessee, United States, St. Jude Children's Research Hospital, 38105 [Not yet recruiting]
  • Houston, Texas, United States, Texas Children's Hospital, 77030 [Recruiting]
  • Zagazig, Al Sharkeya, Egypt, Zagazig University Hospital [Recruiting]
  • Alexandria, Egypt, Alexandria University Hospital, 21131 [Recruiting]
  • Cairo, Egypt, Ain Shams University Hospital, 11566 [Recruiting]
  • Cairo, Egypt, Abu El Rich Hospital, Cairo University Hospital [Recruiting]
  • Paris, France, Hopital Robert-Debré AP-HP, 75019 [Not yet recruiting]
  • Accra, Ghana, Korle-Bu Teaching Hospital Child Health [Recruiting]
  • Kumasi, Ghana, Komfo Anokye Teaching Hospital Research and Development Unit [Recruiting]
  • Firenze, Italy, Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer, 50139 [Recruiting]
  • Napoli, Italy, Seconda Università degli Studi di Napoli, 80138 [Not yet recruiting]
  • Padova, Italy, Azienda Ospedaliera di Padova, 35128 [Recruiting]
  • Nairobi, Kenya, CREATES - Strathmore University Medical Centre [Recruiting]
  • Nairobi, Kenya, Gertrude's Children's Hospital [Recruiting]
  • Nairobi, Kenya, KEMRI - Centre for Respiratory Disease Research (CRDR) [Recruiting]
  • Siaya, Kenya, KEMRI - Centre for Respiratory Disease Research (CRDR) - Siaya Site [Not yet recruiting]
  • Enugu, Enugu State, Nigeria, University of Nigeria Teaching Hospital [Recruiting]
  • Kano, Kano State, Nigeria, Aminu Kano Teaching Hospital [Recruiting]
  • Ibadan, Nigeria, University College Hospital [Recruiting]
  • Kaduna, Nigeria, Barau Dikko Teaching Hospital / Kaduna State University [Recruiting]
  • Lagos, Nigeria, Lagos University Teaching Hospital [Recruiting]
  • Muscat, Oman, Sultan َQaboos University Hospital - Adults/Adolescents [Recruiting]
  • Muscat, Oman, Sultan َQaboos University Hospital - Pediatrics [Recruiting]
  • Riyadh, Saudi Arabia, King Abdullah Children's Specialist Hospital, 14611 [Recruiting]
  • Riyadh, Saudi Arabia, King Saud University [Recruiting]
  • London, Greater London, United Kingdom, Royal London Hospital, E1 1BB [Not yet recruiting]
  • London, Greater London, United Kingdom, Evelina Childrens Hospital, SE1 7EH [Not yet recruiting]
  • London, Greater London, United Kingdom, King's College Hospital, SE5 9NU [Not yet recruiting]
  • London, Greater London, United Kingdom, St Mary's Hospital - Dept of Haematology, W2 1NY [Withdrawn]
Last updated 2022-08-16