Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
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, Baltimore, Baton Rouge, Boston, Chapel Hill, Charleston, Chicago, Detroit, Durham, Houston, Indianapolis, Little Rock, Memphis, Miami, New Orleans, New York, Newark, Oakland, Oklahoma City, Philadelphia, Pittsburgh, Richmond, and The Bronx, United StatesSee the 34 sites
- Phase
- Phase 3
Where in United States
- Children's Healthcare of Atlanta, Scottish RiteAtlanta, United StatesNo longer taking part
- Johns Hopkins University/Sickle Cell Infusion CenterBaltimore, United StatesNo longer taking part
- Our Lady of the Lake Physician Group-Medical OncologyBaton Rouge, United StatesNo longer taking part
- St. Jude Affiliate Clinic Baton RougeBaton Rouge, United StatesNo longer taking part
- Brigham and Women's HospitalBoston, United StatesNo longer taking part
- Brigham and Women's Hospital Research PharmacyBoston, United StatesNo longer taking part
- Dana-Farber Cancer InstituteBoston, United StatesNo longer taking part
- Clinical and Translational Research Center (CTRC)Chapel Hill, United StatesNo longer taking part
- UNC HospitalsChapel Hill, United StatesNo longer taking part
- Medical University of South Carolina - Comprehensive Sickle Cell ClinicCharleston, United StatesNo longer taking part
- Medical University of South Carolina: Investigational Drug Services PharmacyCharleston, United StatesNo longer taking part
- University of Illinois at Chicago Clinical Research CenterChicago, United StatesNo longer taking part
- University of Illinois Hospital and Health Science SystemChicago, United StatesNo longer taking part
- Children's Hospital of MichiganDetroit, United StatesNo longer taking part
- Duke University Medical CenterDurham, United StatesNo longer taking part
- Investigational Drug Service, Duke University HospitalDurham, 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
- Indiana Hemophilia and Thrombosis CenterIndianapolis, United StatesNo longer taking part
- Arkansas Primary Care Clinic, PALittle Rock, United StatesNo longer taking part
- Methodist Comprehensive Sickle Cell ClinicMemphis, United StatesNo longer taking part
- Jackson Memorial HospitalMiami, United StatesNo longer taking part
- Jackson Memorial Hospital (Investigational Drug Services)Miami, United StatesNo longer taking part
- University of MiamiMiami, United StatesNo longer taking part
- University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)Miami, United StatesNo longer taking part
- University Medical Center New OrleansNew Orleans, United StatesNo longer taking part
- Columbia University Medical Center - Herbert Irving PavilionNew York, United StatesNo longer taking part
- Newark Beth Israel Medical CenterNewark, United StatesNo longer taking part
- UCSF Benioff Children's Hospital OaklandOakland, United StatesNo longer taking part
- Lynn Health Science InstituteOklahoma City, United StatesNo longer taking part
- Thomas Jefferson UniversityPhiladelphia, United StatesNo longer taking part
- University of Pittsburgh Medical Center (UPMC)Pittsburgh, United StatesNo longer taking part
- Clinical Research Services Unit- Virginia Commonwealth UniversityRichmond, United StatesNo longer taking part
- Montefiore Einstein Center for Cancer CareThe Bronx, United StatesNo longer taking part
Trial details
- Who runs it
- Pfizer
- Trial code
- NCT03573882
- Record updated
- 18 November 2025
- Original description
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical TrialsRead full original descriptionShow less
An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
Inclusion: Male or female study participants with SCD who participated and received study treatment in Study GBT440-031; Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor; Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1; Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug; Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).
Exclusion: Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent; Female who is breast-feeding or pregnant; Participant withdrew consent from Study GBT440-031; Participant was lost to follow-up from Study GBT440-031; Participant requiring chronic dialysis.