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

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

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

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Oxbryta® (voxelotor) 500mg Tablets

Detailed 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.

• Study Type: Observational
• Enrollment (Estimated): 1000

Contacts and Locations

• Study Contact: Name: Pfizer CT.gov Call Center; Phone Number: 1-800-718-1021; Email: ClinicalTrials.gov_Inquiries@pfizer.com

Study Locations

• United States
  • Alabama
    • Mobile, Alabama, United States, 36617
      • Recruiting
      • University of South Alabama
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Recruiting
      • Phoenix Children's Hospital
  • California
    • La Jolla, California, United States, 92093-0987
      • Recruiting
      • University of California, San Diego
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Stanford Children's Hospital
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital)
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Department of Pediatrics, Hematology section
  • Connecticut
    • Farmington, Connecticut, United States, 06030
      • Recruiting
      • University of Connecticut Health
      • Contact: Sasia Jones; Phone Number: 860-679-7879; Email: sajones@uchc.edu
      • Principal Investigator: Biree Andemariam, MD
    • Farmington, Connecticut, United States, 06030-1163
      • Recruiting
      • University of Connecticut Health
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Recruiting
      • Nemours Alfred I duPont Hospital for Children
    • Wilmington, Delaware, United States, 19803
      • Recruiting
      • Nemours Children's Health, Wilmington
  • Florida
    • Hollywood, Florida, United States, 33023
      • Active, not recruiting
      • Foundation for Sickle Cell Disease Research
    • Jacksonville, Florida, United States, 32207
      • Recruiting
      • Nemours Children's Specialty Care
    • Miami, Florida, United States, 33136
      • Not yet recruiting
      • University of Miami Hospital
  • Georgia
    • Augusta, Georgia, United States, 30912
      • Recruiting
      • Augusta University
      • Contact: Amir Mian, MD; Email: amian@augusta.edu
      • Principal Investigator: Amir Mian, MD
    • Augusta, Georgia, United States, 30912
      • Recruiting
      • Augusta University - Clinical Trials Office (clinic)
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Recruiting
      • University of Illinois Hospital and Health Sciences System
    • Chicago, Illinois, United States, 60612
      • Recruiting
      • University of Illinois at Chicago (UIC) Clinical Research Center
    • Chicago, Illinois, United States, 60612
      • Recruiting
      • University of Illinois at Chicago (UIC) Sickle Cell Center
    • Chicago, Illinois, United States, 60612
      • Recruiting
      • University of Illinois Hospital and Health Sciences System(UI Health)
  • Maryland
    • College Park, Maryland, United States, 21201
      • Recruiting
      • University of Maryland Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
      • Recruiting
      • Boston University Medical Center
  • Mississippi
    • Madison, Mississippi, United States, 39110
      • Recruiting
      • Mississippi Center for Advanced Medicine
      • Contact: Wendy Thomson; Email: wthomson@msadvancedmedicine.com
      • Principal Investigator: Sharon Pennington, MD
  • New York
    • Bronx, New York, United States, 10467
      • Recruiting
      • Montefiore Medical Center
      • Contact: Andrew Crouch; Phone Number: 248-498-4668; Email: andrew.crouch@einsteinmed.org
      • Principal Investigator: Henny Billett, MD
  • North Carolina
    • Charlotte, North Carolina, United States, 28204
      • Recruiting
      • Levine Cancer Institute
    • Durham, North Carolina, United States, 27710
      • Recruiting
      • Duke University Medical Center
    • Durham, North Carolina, United States, 27710
      • Recruiting
      • Duke University Hospital
      • Contact: Stephanie Padrick; Phone Number: 919-684-2406; Email: stephanie.padrick@duke.edu
      • Principal Investigator: Nirmish Shah, MD
    • Greenville, North Carolina, United States, 27834
      • Recruiting
      • ECU Health Medical Center
    • Greenville, North Carolina, United States, 27834-4300
      • Recruiting
      • East Carolina University
    • Greenville, North Carolina, United States, 27834
      • Recruiting
      • ECU Health Medical Center Laboratory
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • Recruiting
      • UPMC Presbyterian
    • Pittsburgh, Pennsylvania, United States, 14213
      • Recruiting
      • UPMC Montefiore Hospital
    • Pittsburgh, Pennsylvania, United States, 15123
      • Recruiting
      • UPMC Sickle Cell Center
    • Pittsburgh, Pennsylvania, United States, 15261
      • Recruiting
      • University of Pittsburgh Medical Center (Upmc)
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina
      • Contact: Karen Hawkins; Phone Number: 843-792-0560; Email: hawkinsk@musc.edu
      • Principal Investigator: Shayla Bergmann, MD
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina Shawn Jenkins Women's and Children's Hospital
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Recruiting
      • St. Jude Children's Research Hospital
      • Principal Investigator: Parul Rai, MD
      • Contact: Twanna Smith; Phone Number: 800-822-6344
  • Texas
    • Austin, Texas, United States, 78723
      • Recruiting
      • Dell Children's Medical Center
      • Contact: Rhea Robinson; Email: rmrobinson@ascension.org
      • Principal Investigator: Alicia Chang, MD
    • Austin, Texas, United States, 78723
      • Recruiting
      • Children's Blood and Cancer Center at Dell Children's Medical Center
    • Houston, Texas, United States, 77030
      • Recruiting
      • University of Texas Health Science Center at Houston
  • Virginia
    • Fairfax, Virginia, United States, 22031
      • Recruiting
      • Inova Schar Cancer Institute
    • Falls Church, Virginia, United States, 22042-2325
      • Recruiting
      • INOVA Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Oxbryta Product Registry	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.; Other Names: Voxelotor; Oxbryta®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from pre-Oxbryta treatment period in Hemoglobin (Hb)		1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in percent Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in absolute Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in bilirubin		1 year before and 1 year after the first dose of Oxbryta
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
Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits		1 year before and 1 year after the first dose of Oxbryta
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
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
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
Incidence and severity of serious adverse events (SAEs)		1 year before and 1 year after the first dose of Oxbryta
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

Collaborators and Investigators

• Sponsor: Pfizer
• Collaborators: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): November 12, 2021
• Primary Completion (Estimated): October 30, 2029
• Study Completion (Estimated): October 30, 2029

Study Registration Dates

• First Submitted: June 4, 2021
• First Submitted That Met QC Criteria: June 10, 2021
• First Posted (Actual): June 18, 2021

Study Record Updates

• Last Update Posted (Actual): April 11, 2024
• Last Update Submitted That Met QC Criteria: April 10, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Registry Sickle Cell Disease
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: GBT440-4R2; C5341019 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No