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

Study point of contact

Jenni Herber
(650) 826-2422
[email protected]
Margaret Tonda, PharmD
(650) 741-7761
[email protected]

Locations

16 United States sites

5 Nigeria sites

4 Egypt sites

3 Italy sites

3 Kenya sites

3 United Kingdom sites

2 Oman sites

2 Ghana sites

2 Saudi Arabia sites

Age

2 to 14 Years

Phase

Phase 3

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

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.

participation requirements

1. Male or female participants with Sickle Cell Anemia (SCA)

2. TCD time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow ≥
170 to < 200cm/sec during the Screening Period 3. Hb ≥ 5.5 and ≤ 10.5 g/dL during screening 4. 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 5. 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

1. Body weight < 5kg at the screening visit 2. 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). 3. More than 10 VOCs within the past 12 months that required hospitalization, emergency room, or clinic visit 4. Stroke resulting in focal neurological deficit; previous silent infarcts are permitted. 5. Known history or findings, including screening magnetic resonance imaging (MRI)/magnetic imaging angiography (MRA) findings, suggestive of significant cerebral vasculopathy (eg, moyamoya or significant vasculopathy) 6. History of seizure disorder (History of febrile seizures is permissible if there have been no seizures within the 12 months prior to randomization). 7. 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 8. 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

Locations

  • Birmingham, Alabama, United States, University of Alabama at Birmingham, 35233 [Not yet recruiting]
  • Washington, District of Columbia, United States, Children's National Medical Center, 20010 [Recruiting]
  • Miami, Florida, United States, University of Miami Medical Center, 33136 [Not yet recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta-Hughes Spalding, 30303 [Not yet recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta-Scottish Rite, 30342 [Recruiting]
  • Chicago, Illinois, United States, Ann & Robert H. Lurie Children's Hospital of Chicago, 60611 [Not yet recruiting]
  • Boston, Massachusetts, United States, Boston Children's Hospital, 02111 [Not yet recruiting]
  • Detroit, Michigan, United States, Wayne State University, 48201 [Not yet recruiting]
  • Jackson, Mississippi, United States, University of Mississippi Medical Center, 39216 [Not yet recruiting]
  • Kansas City, Missouri, United States, Children's Mercy Hospitals & Clinics, 64108 [Not yet recruiting]
  • Saint Louis, Missouri, United States, Washington University School of Medicine, 63110 [Not yet 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 [Not yet recruiting]
  • Zagazig, Al Sharkeya, Egypt, Zagazig University Hospital [Not yet recruiting]
  • Alexandria, Egypt, Alexandria University Hospital, 21131 [Not yet recruiting]
  • Cairo, Egypt, Ain Shams University Hospital, 11566 [Not yet recruiting]
  • Cairo, Egypt, Abu El Rich Hospital, Cairo University Hospital [Not yet recruiting]
  • Accra, Ghana, Korle-Bu Teaching Hospital Child Health [Not yet recruiting]
  • Kumasi, Ghana, Komfo Anokye Teaching Hospital Research and Development Unit [Not yet recruiting]
  • Firenze, Italy, Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer, 50139 [Not yet recruiting]
  • Napoli, Italy, Seconda Università degli Studi di Napoli, 80138 [Not yet recruiting]
  • Padova, Italy, Azienda Ospedaliera di Padova, 35128 [Not yet recruiting]
  • Nairobi, Kenya, CREATES - Strathmore University Medical Centre [Not yet recruiting]
  • Nairobi, Kenya, Gertrude's Children's Hospital [Not yet recruiting]
  • Nairobi, Kenya, KEMRI - Centre for Respiratory Disease Research (CRDR) [Not yet recruiting]
  • Enugu, Enugu State, Nigeria, University of Nigeria Teaching Hospital [Not yet recruiting]
  • Kano, Kano State, Nigeria, Aminu Kano Teaching Hospital [Not yet recruiting]
  • Ibadan, Nigeria, University College Hospital [Not yet recruiting]
  • Kaduna, Nigeria, Barau Dikko Teaching Hospital / Kaduna State University [Not yet recruiting]
  • Lagos, Nigeria, Lagos University Teaching Hospital [Not yet recruiting]
  • Muscat, Oman, Sultan َQaboos University Hospital - Adults/Adolescents [Not yet recruiting]
  • Muscat, Oman, Sultan َQaboos University Hospital - Pediatrics [Not yet recruiting]
  • Riyadh, Saudi Arabia, King Abdullah Children's Specialist Hospital, 14611 [Not yet recruiting]
  • Riyadh, Saudi Arabia, King Saud University [Not yet 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]