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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
This open label extension (OLE), multi-center study will be conducted at approximately 100 clinical sites globally and will be available to eligible participants from study GBT440-031. The study will enroll participants from GBT440-031 (approximately 435) under any of the following conditions: * Participant has completed 72 weeks of treatment regardless of dose selection for GBT440-031 * Dose selection has occurred for GBT440-031 and participant is on non-selected dose on GBT440-031 * GBT440-031 study interim data analysis and/or study modifications have occurred * GBT440-031 study has completed The objective of this open-label extension (OLE) study is to assess the long-term safety and treatment effect of voxelotor in participants who have completed treatment in study GBT440-031, using the following parameters: 1. Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures. 2. Frequency of sickle cell disease (SCD)-related complications. 3. Hemolytic anemia as measured by hematological laboratory parameters (e.g. hemoglobin, reticulocytes and unconjugated bilirubin). All participants will receive daily voxelotor treatment. Participants may receive study drug as long they continue to receive clinical benefit which outweighs risk as determined by the Investigator and/or until the participant has access to voxelotor from an alternative source (i.e., commercialization or through a managed access program).
Age
12 - 65 years
Sex
ALL
Healthy Volunteers
No
Arkansas Primary Care Clinic, PA
Little Rock, Arkansas, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Jackson Memorial Hospital (Investigational Drug Services)
Miami, Florida, United States
Jackson Memorial Hospital
Miami, Florida, United States
University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)
Miami, Florida, United States
University of Miami
Miami, Florida, United States
Children's Healthcare of Atlanta - Scottish Rite
Atlanta, Georgia, United States
University of Illinois at Chicago Clinical Research Center
Chicago, Illinois, United States
University of Illinois Hospital and Health Science System
Chicago, Illinois, United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
Start Date
June 6, 2018
Primary Completion Date
November 12, 2024
Completion Date
November 12, 2024
Last Updated
November 18, 2025
179
ACTUAL participants
Voxelotor
DRUG
Lead Sponsor
Pfizer
NCT05170412
NCT06526117
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.
View ClinicalTrials.gov Terms and ConditionsNCT06665997