Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
- Registration Number
- NCT03573882
- Lead Sponsor
- Pfizer
- Brief Summary
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
- Detailed Description
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).
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 179
- 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).
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Voxelotor 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).
- Primary Outcome Measures
Name Time Method Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 Five Years Safety based on Adverse Events
Frequency of sickle cell-related complications Five Years Frequency of SCD-related complications with long-term dosing with voxelotor.
- Secondary Outcome Measures
Name Time Method Response in Hemolytic Anemia Five Years Measured by Hemoglobin, Bilirubin, and Reticulocyte counts
Trial Locations
- Locations (65)
Children's Healthcare of Atlanta at Hughes Spalding
๐บ๐ธAtlanta, Georgia, United States
University of Illinois Hospital and Health Science System
๐บ๐ธChicago, Illinois, United States
Dana Farber Cancer Institute
๐บ๐ธBoston, Massachusetts, United States
University of Illinois at Chicago Clinical Research Center
๐บ๐ธChicago, Illinois, United States
Brigham and Women's Hospital
๐บ๐ธBoston, Massachusetts, United States
Brigham and Women's Hospital Research Pharmacy
๐บ๐ธBoston, Massachusetts, United States
University of Pittsburgh Medical Center (UPMC)
๐บ๐ธPittsburgh, Pennsylvania, United States
Jackson Memorial Hospital (Investigational Drug Services)
๐บ๐ธMiami, Florida, United States
Jackson Memorial Hospital
๐บ๐ธMiami, Florida, United States
University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)
๐บ๐ธMiami, Florida, United States
University of Miami
๐บ๐ธMiami, Florida, United States
Duke University Medical Center
๐บ๐ธDurham, North Carolina, United States
Investigational Drug Service, Duke University Hospital
๐บ๐ธDurham, North Carolina, United States
Barts Health NHS Trust
๐ฌ๐งLondon, Greater London, United Kingdom
Imperial College Healthcare NHS Trust, Hammersmith Hospital
๐ฌ๐งLondon, United Kingdom
McMillan Cancer Centre
๐ฌ๐งLondon, United Kingdom
Azienda Ospedaliera di Padova
๐ฎ๐นPadova, Padova Veneto, Italy
Toronto General Hospital-University Health Network
๐จ๐ฆToronto, Ontario, Canada
Hรดpital Europรฉen Georges Pompidou
๐ซ๐ทParis, รle-de-france, France
Adana Acibadem Hospital
๐น๐ทAdana, Turkey
KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research
๐ฐ๐ชNairobi, Kenya
Texas Children's Hospital - Investigational Pharmacy
๐บ๐ธHouston, Texas, United States
Erciyes Universitesi Tip Fakultesi Hastanesi
๐น๐ทIstanbul, ฤฐฬstanbul, Turkey
Homerton University Hospital NHS Foundation Trust
๐ฌ๐งLondon, Greater London, United Kingdom
Hรดpital Europรฉen Georges Pompidou - Medecine Interne
๐ซ๐ทParis, France
Texas Children's Hospital
๐บ๐ธHouston, Texas, United States
Erciyes Universitesi Tip Fakultesi
๐น๐ทKayseri, Turkey
Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi
๐น๐ทMersin, Turkey
Guys and St. Thomas Hospital NHS Foundation Trust
๐ฌ๐งGreat Maze Pond, London, United Kingdom
King's College Hospital
๐ฌ๐งLondon, United Kingdom
Nini Hospital
๐ฑ๐งTripoli, Lebanon
Arkansas Primary Care Clinic, PA
๐บ๐ธLittle Rock, Arkansas, United States
UCSF Benioff Children's Hospital Oakland
๐บ๐ธOakland, California, United States
Children's Healthcare of Atlanta - Scottish Rite
๐บ๐ธAtlanta, Georgia, United States
Indiana Hemophilia and Thrombosis Center
๐บ๐ธIndianapolis, Indiana, United States
The Johns Hopkins Hospital
๐บ๐ธBaltimore, Maryland, United States
Johns Hopkins University/Sickle Cell Infusion Center
๐บ๐ธBaltimore, Maryland, United States
Montefiore - Einstein Center for Cancer Care
๐บ๐ธBronx, New York, United States
Columbia University Medical Center - Herbert Irving Pavilion
๐บ๐ธNew York, New York, United States
Guys and STt Thomas NHS Foundation Trust
๐ฌ๐งGreat Maze Pond, London, United Kingdom
Newark Beth Israel Medical Center
๐บ๐ธNewark, New Jersey, United States
ErasmusMC
๐ณ๐ฑRotterdam, Netherlands
Sultan Qaboos University Hospital
๐ด๐ฒMuscat, Oman
The Egyptian Thalassemia Association ( E.T.A)
๐ช๐ฌCairo, Egypt
Methodist Comprehensive Sickle Cell Clinic
๐บ๐ธMemphis, Tennessee, United States
Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
๐ช๐ฌAlexandria, Egypt
St. Jude Affiliate Clinic Baton Rouge
๐บ๐ธBaton Rouge, Louisiana, United States
American University of Beirut
๐ฑ๐งBeirut, Lebanon
Abu El Rich Hospital,Cairo University Hospital
๐ช๐ฌCairo, Egypt
Ain Shams University Hospital
๐ช๐ฌCairo, Egypt
Our Lady of the Lake Physician Group-Medical Oncology
๐บ๐ธBaton Rouge, Louisiana, United States
Alexandria Clinical Research Center, Faculty of Medicine
๐ช๐ฌAlexandria, Egypt
Zagazig University Hospital
๐ช๐ฌAlsharkia, Egypt
Centres for Disease Control and Prevention
๐ฐ๐ชSiaya, Kisumu, Kenya
Gertrude's Children's Hospital
๐ฐ๐ชNairobi, Kenya
Academic Medical Center(AMC)
๐ณ๐ฑAmsterdam, Netherlands
University Medical Center New Orleans
๐บ๐ธNew Orleans, Louisiana, United States
Children's Hospital of Michigan
๐บ๐ธDetroit, Michigan, United States
UNC Hospitals
๐บ๐ธChapel Hill, North Carolina, United States
Clinical and Translational Research Center (CTRC)
๐บ๐ธChapel Hill, North Carolina, United States
Lynn Health Science Institute
๐บ๐ธOklahoma City, Oklahoma, United States
Medical University of South Carolina - Comprehensive Sickle Cell Clinic
๐บ๐ธCharleston, South Carolina, United States
Medical University of South Carolina: Investigational Drug Services Pharmacy
๐บ๐ธCharleston, South Carolina, United States
Clinical Research Services Unit- Virginia Commonwealth University
๐บ๐ธRichmond, Virginia, United States
Thomas Jefferson University
๐บ๐ธPhiladelphia, Pennsylvania, United States