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

Last updated: November 20, 2024
Sponsor: Pfizer
Overall Status: Terminated

Phase

N/A

Condition

Sickle Cell Disease

Red Blood Cell Disorders

Treatment

Oxbryta® (voxelotor) 500mg Tablets

Clinical Study ID

NCT04930445
GBT440-4R2
C5341019
  • Ages > 4
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

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) perlocal regulations, or pediatric participants (aged 4 to <12 years) withparental/guardian consent per Institutional Review Board (IRB) policy andrequirements, 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

Exclusion Criteria:

  • Participants meeting any of the following criteria will not be eligible for studyenrollment:
  1. Current participation in an investigation clinical trial or expanded accessprogram, in which the participant may be receiving voxelotor treatment.

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

Study Design

Total Participants: 262
Treatment Group(s): 1
Primary Treatment: Oxbryta® (voxelotor) 500mg Tablets
Phase:
Study Start date:
February 04, 2022
Estimated Completion Date:
October 10, 2024

Study 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.

Connect with a study center

  • University of South Alabama

    Mobile, Alabama 36617
    United States

    Site Not Available

  • Phoenix Children's Hospital

    Phoenix, Arizona 85016
    United States

    Site Not Available

  • University of California, San Diego

    La Jolla, California 92093-0987
    United States

    Site Not Available

  • Center for Inherited Blood Disorders

    Orange, California 92868
    United States

    Site Not Available

  • Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital)

    Palo Alto, California 94304
    United States

    Site Not Available

  • Department of Pediatrics, Hematology section

    Palo Alto, California 94304
    United States

    Site Not Available

  • Stanford Children's Health- Lucille Packard Children's Hospital Stanford

    Palo Alto, California 94304
    United States

    Active - Recruiting

  • Stanford Children's Hospital

    Palo Alto, California 94304
    United States

    Site Not Available

  • University of Connecticut Health

    Farmington, Connecticut 06030-1163
    United States

    Site Not Available

  • Nemours Alfred I duPont Hospital for Children

    Wilmington, Delaware 19803
    United States

    Site Not Available

  • Nemours Children's Health, Wilmington

    Wilmington, Delaware 19803
    United States

    Site Not Available

  • Foundation for Sickle Cell Disease Research

    Hollywood, Florida 33023
    United States

    Site Not Available

  • Nemours Children's Specialty Care

    Jacksonville, Florida 32207
    United States

    Site Not Available

  • University of Miami Hospital

    Miami, Florida 33136
    United States

    Site Not Available

  • Augusta University

    Augusta, Georgia 30912
    United States

    Site Not Available

  • Augusta University - Clinical Trials Office (clinic)

    Augusta, Georgia 30912
    United States

    Site Not Available

  • University of Illinois Hospital and Health Sciences System

    Chicago, Illinois 60612
    United States

    Site Not Available

  • University of Illinois Hospital and Health Sciences System(UI Health)

    Chicago, Illinois 60612
    United States

    Site Not Available

  • University of Illinois at Chicago (UIC) Clinical Research Center

    Chicago, Illinois 60612
    United States

    Site Not Available

  • University of Illinois at Chicago (UIC) Sickle Cell Center

    Chicago, Illinois 60612
    United States

    Site Not Available

  • University of Maryland Medical Center

    College Park, Maryland 21201
    United States

    Site Not Available

  • Boston University Medical Center

    Boston, Massachusetts 02118
    United States

    Site Not Available

  • Mississippi Center for Advanced Medicine

    Madison, Mississippi 39110
    United States

    Site Not Available

  • Newark Beth Israel Medical Center

    Newark, New Jersey 07112
    United States

    Site Not Available

  • Montefiore Medical Center

    Bronx, New York 10467
    United States

    Site Not Available

  • Levine Cancer Institute

    Charlotte, North Carolina 28204
    United States

    Site Not Available

  • Levine Cancer Institute - Atrium Health

    Charlotte, North Carolina 28204
    United States

    Active - Recruiting

  • Duke University Hospital

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27710
    United States

    Site Not Available

  • ECU Health Medical Center

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • ECU Health Medical Center Laboratory

    Greenville, North Carolina 27834
    United States

    Site Not Available

  • East Carolina University

    Greenville, North Carolina 27834-4300
    United States

    Site Not Available

  • East Carolina University - Brody School of Medicine

    Greenville, North Carolina 27834
    United States

    Active - Recruiting

  • UPMC Montefiore Hospital

    Pittsburgh, Pennsylvania 14213
    United States

    Site Not Available

  • UPMC Presbyterian

    Pittsburgh, Pennsylvania 15213
    United States

    Site Not Available

  • UPMC Sickle Cell Center

    Pittsburgh, Pennsylvania 15123
    United States

    Site Not Available

  • University of Pittsburgh Medical Center (UPMC)

    Pittsburgh, Pennsylvania 15261
    United States

    Site Not Available

  • Medical University of South Carolina

    Charleston, South Carolina 29425
    United States

    Site Not Available

  • Medical University of South Carolina Shawn Jenkins Women's and Children's Hospital

    Charleston, South Carolina 29425
    United States

    Site Not Available

  • St. Jude Children's Research Hospital

    Memphis, Tennessee 38105
    United States

    Site Not Available

  • Children's Blood and Cancer Center at Dell Children's Medical Center

    Austin, Texas 78723
    United States

    Site Not Available

  • Dell Children's Medical Center

    Austin, Texas 78723
    United States

    Site Not Available

  • University of Texas Health Science Center at Houston

    Houston, Texas 77030
    United States

    Site Not Available

  • Inova Schar Cancer Institute

    Fairfax, Virginia 22031
    United States

    Site Not Available

  • INOVA Health

    Falls Church, Virginia 22042-2325
    United States

    Site Not Available

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