Clinical trial NCT04930445: Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD
Official title: An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Sickle Cell Disease
Keywords: Registry Sickle Cell Disease
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-06-18
Enrollment: 262
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
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.

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

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change from pre-Oxbryta treatment period in Hemoglobin (Hb)
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Change from pre-Oxbryta treatment period in percent Reticulocytes
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Change from pre-Oxbryta treatment period in absolute Reticulocytes
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Change from pre-Oxbryta treatment period in bilirubin
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Incidence of significant SCD-related clinical events
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Change from pre-Oxbryta treatment period in incidence of emergency department (ED) visits
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Change from pre-Oxbryta treatment period in incidence of hospitalizations (including total length of stay, and time in intensive care unit [ICU], if applicable)
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Change from pre-Oxbryta treatment period in incidence of red blood cell transfusions
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Incidence and severity of serious adverse events (SAEs)
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
measure: Incidence and severity of adverse events (AEs) of interest
timeFrame: 1 year before and 1 year after the first dose of Oxbryta
Secondary outcomes:
Other outcomes:
Locations:
city: Mobile
country: United States
facility: University of South Alabama
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: La Jolla
country: United States
facility: University of California, San Diego
state: California
city: Orange
country: United States
facility: Center for Inherited Blood Disorders
state: California
city: Palo Alto
country: United States
facility: Bass Center for Childhood Cancer and Blood Disorders (Stanford Lucile Packard Children's Hospital)
state: California
city: Palo Alto
country: United States
facility: Department of Pediatrics, Hematology section
state: California
city: Palo Alto
country: United States
facility: Stanford Children's Hospital
state: California
city: Farmington
country: United States
facility: University of Connecticut Health
state: Connecticut
city: Farmington
country: United States
facility: University of Connecticut Health
state: Connecticut
city: Wilmington
country: United States
facility: Nemours Alfred I duPont Hospital for Children
state: Delaware
city: Wilmington
country: United States
facility: Nemours Children's Health, Wilmington
state: Delaware
city: Hollywood
country: United States
facility: Foundation for Sickle Cell Disease Research
state: Florida
city: Jacksonville
country: United States
facility: Nemours Children's Specialty Care
state: Florida
city: Miami
country: United States
facility: University of Miami Hospital
state: Florida
city: Augusta
country: United States
facility: Augusta University - Clinical Trials Office (clinic)
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Chicago
country: United States
facility: University of Illinois at Chicago (UIC) Clinical Research Center
state: Illinois
city: Chicago
country: United States
facility: University of Illinois at Chicago (UIC) Sickle Cell Center
state: Illinois
city: Chicago
country: United States
facility: University of Illinois Hospital and Health Sciences System(UI Health)
state: Illinois
city: Chicago
country: United States
facility: University of Illinois Hospital and Health Sciences System
state: Illinois
city: College Park
country: United States
facility: University of Maryland Medical Center
state: Maryland
city: Boston
country: United States
facility: Boston University Medical Center
state: Massachusetts
city: Madison
country: United States
facility: Mississippi Center for Advanced Medicine
state: Mississippi
city: Newark
country: United States
facility: Newark Beth Israel Medical Center
state: New Jersey
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Charlotte
country: United States
facility: Levine Cancer Institute
state: North Carolina
city: Durham
country: United States
facility: Duke University Hospital
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Greenville
country: United States
facility: ECU Health Medical Center Laboratory
state: North Carolina
city: Greenville
country: United States
facility: ECU Health Medical Center
state: North Carolina
city: Pittsburgh
country: United States
facility: UPMC Montefiore Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Sickle Cell Center
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Presbyterian
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center (UPMC)
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina Shawn Jenkins Women's and Children's Hospital
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Austin
country: United States
facility: Children's Blood and Cancer Center at Dell Children's Medical Center
state: Texas
city: Austin
country: United States
facility: Dell Children's Medical Center
state: Texas
city: Houston
country: United States
facility: University of Texas Health Science Center at Houston
state: Texas
city: Fairfax
country: United States
facility: Inova Schar Cancer Institute
state: Virginia
city: Falls Church
country: United States
facility: INOVA Health
state: Virginia
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: