Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
NCT ID: NCT03573882
Last Updated: 2025-11-18
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
179 participants
INTERVENTIONAL
2018-06-06
2024-11-12
Brief Summary
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Detailed Description
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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).
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Voxelotor
Participants will receive voxelotor (GBT440) at the highest dose (either 900 mg or 1500 mg) deemed safe by the Data Safety Monitoring Board (DSMB).
Voxelotor
300mg or 500mg Tablet, Oral, With or Without Food
Interventions
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Voxelotor
300mg or 500mg Tablet, Oral, With or Without Food
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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 Criteria
* Participant withdrew consent from Study GBT440-031.
* Participant was lost to follow-up from Study GBT440-031.
* Participant requiring chronic dialysis.
* 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.
12 Years
65 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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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
St. Jude Affiliate Clinic Baton Rouge
Baton Rouge, Louisiana, United States
Our Lady of the Lake Physician Group-Medical Oncology
Baton Rouge, Louisiana, United States
University Medical Center New Orleans
New Orleans, Louisiana, United States
Johns Hopkins University/Sickle Cell Infusion Center
Baltimore, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Brigham and Women's Hospital Research Pharmacy
Boston, Massachusetts, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Newark Beth Israel Medical Center
Newark, New Jersey, United States
Columbia University Medical Center - Herbert Irving Pavilion
New York, New York, United States
Montefiore - Einstein Center for Cancer Care
The Bronx, New York, United States
UNC Hospitals
Chapel Hill, North Carolina, United States
Clinical and Translational Research Center (CTRC)
Chapel Hill, North Carolina, United States
Duke University Medical Center
Durham, North Carolina, United States
Investigational Drug Service, Duke University Hospital
Durham, North Carolina, United States
Lynn Health Science Institute
Oklahoma City, Oklahoma, United States
Thomas Jefferson University
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina - Comprehensive Sickle Cell Clinic
Charleston, South Carolina, United States
Medical University of South Carolina: Investigational Drug Services Pharmacy
Charleston, South Carolina, United States
Methodist Comprehensive Sickle Cell Clinic
Memphis, Tennessee, United States
Texas Children's Hospital - Investigational Pharmacy
Houston, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
Clinical Research Services Unit- Virginia Commonwealth University
Richmond, Virginia, United States
Toronto General Hospital-University Health Network
Toronto, Ontario, Canada
Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
Alexandria, , Egypt
Alexandria Clinical Research Center, Faculty of Medicine
Alexandria, , Egypt
Zagazig University Hospital
Alsharkia, , Egypt
Abu El Rich Hospital,Cairo University Hospital
Cairo, , Egypt
Ain Shams University Hospital
Cairo, , Egypt
The Egyptian Thalassemia Association ( E.T.A)
Cairo, , Egypt
Hôpital Européen Georges Pompidou - Medecine Interne
Paris, , France
Azienda Ospedaliera di Padova
Padua, Padova Veneto, Italy
Centres for Disease Control and Prevention
Siaya, Kisumu County, Kenya
KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research
Nairobi, , Kenya
Gertrude's Children's Hospital
Nairobi, , Kenya
American University of Beirut
Beirut, , Lebanon
Nini Hospital
Tripoli, , Lebanon
Academic Medical Center(AMC)
Amsterdam, , Netherlands
ErasmusMC
Rotterdam, , Netherlands
Sultan Qaboos University Hospital
Muscat, , Oman
Adana Acibadem Hospital
Adana, , Turkey (Türkiye)
Erciyes Universitesi Tip Fakultesi
Kayseri, , Turkey (Türkiye)
Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi
Mersin, , Turkey (Türkiye)
Barts Health NHS Trust
London, Greater London, United Kingdom
Homerton University Hospital NHS Foundation Trust
London, Greater London, United Kingdom
Guys and St. Thomas Hospital NHS Foundation Trust
Great Maze Pond, London, United Kingdom
King's College Hospital
London, , United Kingdom
Imperial College Healthcare NHS Trust, Hammersmith Hospital
London, , United Kingdom
McMillan Cancer Centre
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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GBT440-034
Identifier Type: -
Identifier Source: org_study_id
C5341022
Identifier Type: OTHER
Identifier Source: secondary_id
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