Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD

Sickle Cell Disease Clinical Trial

— PROSPECT  

Official title:

An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04930445
• Other study ID #: GBT440-4R2
• Secondary ID: C5341019
• Status: Recruiting
• Start date: November 12, 2021
• Est. completion date: October 30, 2029

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.

Description:

The study will be conducted at approximately 45 sites in the United States.

This registry is an observational study to evaluate the effects of Oxbryta in individuals with SCD. Any participant who is currently taking Oxbryta, or has been prescribed and will initiate treatment with Oxbryta, is eligible to participate. Eligible participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. This study will collect data that are recorded in the participants' medical records and other secondary data sources. Study data will be collected at regular intervals and entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff. Participants will be considered to be on study for up to 5 years after their first dose of Oxbryta treatment, or until they withdraw their consent to participate, or are discontinued from the study. Treatment, including interruptions and restarting treatment, will continue at the discretion of the treating physician, and there are no pre-defined treatment requirements. Participants may receive any additional medications prescribed by their treating physician, or have any medical interventions that are deemed appropriate by the treating physician or study doctor. The participant or treating physician may discontinue Oxbryta at any time. Participants who discontinue treatment with Oxbryta earlier than 5 years will continue to be followed on study to collect clinical and quality of life (QoL) outcomes for up to 5 years after their first dose of Oxbryta treatment. Participant safety and tolerability will be assessed throughout the study data collection period by the study doctor and reported to the Sponsor.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1000
• Est. completion date: October 30, 2029
• Est. primary completion date: October 30, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

Inclusion Criteria:

• Participants who meet all the following criteria will be eligible for enrollment:

1. Willing and able to provide written informed consent (aged = 18 years), parental/ guardian consent and participant assent (aged = 12 to <18 years) per local regulations, or pediatric participants (aged 4 to <12 years) with parental/guardian consent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines

2. Male or female participants with documented diagnosis of sickle cell disease (all genotypes)

3. Undergoing treatment with Oxbryta according to the Oxbryta USPI

Exclusion Criteria:

• Participants meeting any of the following criteria will not be eligible for study enrollment:

1. Current participation in an investigation clinical trial or expanded access program, in which the participant may be receiving voxelotor treatment.

2. Medical, psychological, or behavioral condition that, in the opinion of the study doctor, would confound or interfere with evaluation of safety and/or effectiveness of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Oxbryta® (voxelotor) 500mg Tablets
Participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. There are no pre-defined treatment requirements.

Locations

Country	Name	City	State
United States	Augusta University	Augusta	Georgia
United States	Augusta University - Clinical Trials Office (clinic)	Augusta	Georgia
United States	Children's Blood and Cancer Center at Dell Children's Medical Center	Austin	Texas
United States	Dell Children's Medical Center	Austin	Texas
United States	Boston University Medical Center	Boston	Massachusetts
United States	Montefiore Medical Center	Bronx	New York
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Medical University of South Carolina Shawn Jenkins Women's and Children's Hospital	Charleston	South Carolina
United States	Levine Cancer Institute	Charlotte	North Carolina
United States	University of Illinois at Chicago (UIC) Clinical Research Center	Chicago	Illinois
United States	University of Illinois at Chicago (UIC) Sickle Cell Center	Chicago	Illinois
United States	University of Illinois Hospital and Health Sciences System	Chicago	Illinois
United States	University of Illinois Hospital and Health Sciences System(UI Health)	Chicago	Illinois
United States	University of Maryland Medical Center	College Park	Maryland
United States	Duke University Hospital	Durham	North Carolina
United States	Duke University Medical Center	Durham	North Carolina
United States	Inova Schar Cancer Institute	Fairfax	Virginia
United States	INOVA Health	Falls Church	Virginia
United States	University of Connecticut Health	Farmington	Connecticut
United States	University of Connecticut Health	Farmington	Connecticut
United States	East Carolina University	Greenville	North Carolina
United States	ECU Health Medical Center	Greenville	North Carolina
United States	ECU Health Medical Center Laboratory	Greenville	North Carolina
United States	Foundation for Sickle Cell Disease Research	Hollywood	Florida
United States	University of Texas Health Science Center at Houston	Houston	Texas
United States	Nemours Children's Specialty Care	Jacksonville	Florida
United States	University of California, San Diego	La Jolla	California
United States	Mississippi Center for Advanced Medicine	Madison	Mississippi
United States	St. Jude Children's Research Hospital	Memphis	Tennessee
United States	University of Miami Hospital	Miami	Florida
United States	University of South Alabama	Mobile	Alabama
United States	Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital)	Palo Alto	California
United States	Department of Pediatrics, Hematology section	Palo Alto	California
United States	Stanford Children's Hospital	Palo Alto	California
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	University of Pittsburgh Medical Center (UPMC)	Pittsburgh	Pennsylvania
United States	UPMC Montefiore Hospital	Pittsburgh	Pennsylvania
United States	UPMC Presbyterian	Pittsburgh	Pennsylvania
United States	UPMC Sickle Cell Center	Pittsburgh	Pennsylvania
United States	Nemours Alfred I duPont Hospital for Children	Wilmington	Delaware
United States	Nemours Children's Health, Wilmington	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from pre-Oxbryta treatment period in Hemoglobin (Hb)		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in percent Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in absolute Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in bilirubin		1 year before and 1 year after the first dose of Oxbryta
Primary	Incidence of significant SCD-related clinical events	Such as vaso-occlusive crisis (VOC), acute chest syndrome (ACS), priapism, cerebral infarcts, transient ischemic attack (TIA), leg ulcers, measures of cardiac function and pulmonary hypertension (PH)	1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of emergency department (ED) visits		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of hospitalizations (including total length of stay, and time in intensive care unit [ICU], if applicable)		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of red blood cell transfusions		1 year before and 1 year after the first dose of Oxbryta
Primary	Incidence and severity of serious adverse events (SAEs)		1 year before and 1 year after the first dose of Oxbryta
Primary	Incidence and severity of adverse events (AEs) of interest	Such as Rash, Diarrhea, Headache, AEs leading to Oxbryta dose modification or discontinuation	1 year before and 1 year after the first dose of Oxbryta

External Links

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