Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
NCT ID: NCT04218084
Last Updated: 2025-06-13
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
236 participants
INTERVENTIONAL
2020-11-11
2024-11-06
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Voxelotor
Voxelotor 1500mg or equivalent daily as a tablet, dispersible tablet, or as powder for oral suspension.
Voxelotor
Participants are randomized 1:1 to receive voxelotor or placebo.
Placebo
Matching placebo.
Placebo
Matching placebo.
Interventions
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Voxelotor
Participants are randomized 1:1 to receive voxelotor or placebo.
Placebo
Matching placebo.
Eligibility Criteria
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Inclusion Criteria
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.
Exclusion Criteria
2. Hospitalization for VOC or acute chest syndrome (ACS) within the 14 days prior to execution of informed consent/assent
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 suggestive of significant cerebral vasculopathy
6. History of seizure disorder
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
2 Years
14 Years
ALL
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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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
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
Charleston, South Carolina, United States
Texas Children's Hospital- Wallace Tower
Houston, Texas, United States
Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
Alexandria, , Egypt
Zagazig University Hospital
Alexandria, , Egypt
Abu El Rich Hospital, Cairo University Hospital
Cairo, , Egypt
Ain Shams University Hospital-Clinical Research Center (MASRI)
Cairo, , Egypt
Department of Child Health, University of Ghana Medical School, College of Health Sciences
Accra, , Ghana
Komfo Anokye Teaching Hospital
Kumasi, , Ghana
Azienda Ospedaliera Universitaria (AOU) Meyer
Florence, , Italy
Azienda Ospedaliera Universitaria (A.O.U.) "Luigi Vanvitelli"
Napoli, , Italy
Azienda Ospedaliera Universita (AOU) Padova
Padua, , Italy
Kemri/Crdr,Siaya,Kemri Clinical Research Annex
Kisumu, Siaya County, Kenya
KEMRI CRDR Clinical Research Annex
Nairobi, , Kenya
Strathmore University Medical Centre
Nairobi, , Kenya
Gertrude's Children Hospital
Nairobi, , Kenya
University of Nigeria Teaching Hospital
Enugu, , Nigeria
College of Medicine, University of Ibadan
Ibadan, , Nigeria
Barau Dikko Teaching/ Kaduna State University
Kaduna, , Nigeria
Aminu Kano Teaching Hospital
Kano, , Nigeria
Lagos University Teaching Hospital
Lagos, , Nigeria
Sultan Qaboos University Hospital
Muscat, , Oman
King Abdullah International Medical Research Center (KAIMR) Ministry of National Guard - Health
Riyadh, , Saudi Arabia
Barts Health NHS Trust
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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2017-000903-26
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
C5341021
Identifier Type: OTHER
Identifier Source: secondary_id
GBT440-032
Identifier Type: -
Identifier Source: org_study_id
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