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NCT03573882 Clinical Trial

Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031

Sickle Cell Disease Clinical Trial

— 034OLE  

Official title:
An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT03573882
Other study ID # GBT440-034
Secondary ID C5341022
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date June 6, 2018
Est. completion date December 31, 2024

Study information
Verified date March 2024
Source Pfizer
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials

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 information / eligibility
Status Active, not recruiting
Enrollment 179
Est. completion date December 31, 2024
Est. primary completion date December 31, 2024
Accepts healthy volunteers No
Gender All
Age group 12 Years to 65 Years
Eligibility Inclusion Criteria: - 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 Criteria: - 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

Related Conditions & MeSH terms

Intervention

Drug:
• Voxelotor
300mg or 500mg Tablet, Oral, With or Without Food

Locations
Country Name City State
Canada Toronto General Hospital-University Health Network Toronto Ontario
Egypt Alexandria Clinical Research Center, Faculty of Medicine Alexandria
Egypt Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University 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
France Hôpital Européen Georges Pompidou - Medecine Interne Paris
Italy Azienda Ospedaliera di Padova Padova Padova Veneto
Kenya Gertrude's Children's Hospital Nairobi
Kenya KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research Nairobi
Kenya Centres for Disease Control and Prevention Siaya Kisumu
Lebanon American University of Beirut Beirut
Lebanon Nini Hospital Tripoli
Netherlands Academic Medical Center(AMC) Amsterdam
Netherlands ErasmusMC Rotterdam
Oman Sultan Qaboos University Hospital Muscat
Turkey Adana Acibadem Hospital Adana
Turkey Erciyes Universitesi Tip Fakultesi Kayseri
Turkey Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi Mersin
United Kingdom Guys and STt Thomas NHS Foundation Trust Great Maze Pond London
United Kingdom Barts Health NHS Trust London Greater London
United Kingdom Homerton University Hospital NHS Foundation Trust London Greater London
United Kingdom Imperial College Healthcare NHS Trust, Hammersmith Hospital London
United Kingdom King's College Hospital London
United Kingdom McMillan Cancer Centre London
United States Children's Healthcare of Atlanta - Scottish Rite Atlanta Georgia
United States Children's Healthcare of Atlanta - Scottish Rite Atlanta Georgia
United States Children's Healthcare of Atlanta at Hughes Spalding Atlanta Georgia
United States Johns Hopkins University/Sickle Cell Infusion Center Baltimore Maryland
United States Our Lady of the Lake Physician Group-Medical Oncology Baton Rouge Louisiana
United States St. Jude Affiliate Clinic Baton Rouge Baton Rouge Louisiana
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 Montefiore - Einstein Center for Cancer Care Bronx New York
United States Clinical and Translational Research Center (CTRC) Chapel Hill North Carolina
United States UNC Hospitals Chapel Hill North Carolina
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 University of Illinois at Chicago Clinical Research Center Chicago Illinois
United States University of Illinois Hospital and Health Science System Chicago Illinois
United States Children's Hospital of Michigan Detroit Michigan
United States Duke University Medical Center Durham North Carolina
United States Investigational Drug Service, Duke University Hospital Durham North Carolina
United States Texas Children's Hospital Houston Texas
United States Texas Children's Hospital - Investigational Pharmacy Houston Texas
United States Indiana Hemophilia and Thrombosis Center Indianapolis Indiana
United States Arkansas Primary Care Clinic, PA Little Rock Arkansas
United States Methodist Comprehensive Sickle Cell Clinic Memphis Tennessee
United States Jackson Memorial Hospital Miami Florida
United States Jackson Memorial Hospital (Investigational Drug Services) Miami Florida
United States University of Miami Miami Florida
United States University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services) Miami Florida
United States University Medical Center New Orleans New Orleans Louisiana
United States Columbia University Medical Center - Herbert Irving Pavilion New York New York
United States Newark Beth Israel Medical Center Newark New Jersey
United States UCSF Benioff Children's Hospital Oakland Oakland California
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 Clinical Research Services Unit- Virginia Commonwealth University Richmond Virginia

Sponsors (1)
Lead Sponsor Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Canada,  Egypt,  France,  Italy,  Kenya,  Lebanon,  Netherlands,  Oman,  Turkey,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 Safety based on Adverse Events Five Years
Primary Frequency of sickle cell-related complications Frequency of SCD-related complications with long-term dosing with voxelotor. Five Years
Secondary Response in Hemolytic Anemia Measured by Hemoglobin, Bilirubin, and Reticulocyte counts Five Years
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