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
• 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
• 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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

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

External Links

•   To obtain contact information for a study center near you, click here.