Retrospective Real World Oxbryta® Data Collection and Analysis Study
- Registration Number
- NCT04930328
- Lead Sponsor
- Pfizer
- Brief Summary
The aim of this study is to collect and analyze retrospective data on Oxbryta in a real-world setting. This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300 patients with SCD who have been treated with Oxbryta as part of their usual care. Any patient with SCD who received Oxbryta treatment for at least 2 weeks as part of their usual care according to the Oxbryta US Prescribing Information (USPI) is eligible to participate. Study data from 1 year before and up to 1 year after the first dose of Oxbryta will be entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff.
- Detailed Description
The following are categories of interest in patients with SCD treated with Oxbryta:
* Clinical outcomes, as assessed by clinical and laboratory assessments of hematological parameters and end organ damage, and incidence of significant clinical events
* Healthcare resource utilization
* Health-related quality of life (HRQoL), as assessed by patient-reported outcome (PRO) measures and clinician-reported outcomes (ClinRO)
The safety objective is to assess the safety and tolerability of Oxbryta.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 216
Patients who meet all the following criteria will be eligible for inclusion in this study:
- Willing and able to provide written informed consent (ages greater or equal to 18 years) or parental/guardian consent and patient assent (age <18 years), as required by the IRB or institution or IRB, per local regulations
- Male or female patients with documented diagnosis of SCD (all genotypes)
- Have been treated with Oxbryta for at least 2 weeks, according to the Oxbryta USPI
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Retrospective Data Collection Oxbryta® (voxelotor) 500-mg Tablets Retrospective Data Collection
- Primary Outcome Measures
Name Time Method Incidence of significant SCD-related clinical events 1 year before and 1 year after the first dose of Oxbryta Such as vaso-occlusive crisis (VOC), acute chest syndrome (ACS), priapism, cerebral infarcts, transient ischemic attack (TIA), leg ulcers, and measures of cardiac function and pulmonary hypertension (PH)
Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits 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 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 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 adverse events (AEs) of interest 1 year before and 1 year after the first dose of Oxbryta Such as Rash, Diarrhea, Headache, AEs leading to Oxbryta dose modification or discontinuation
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 Bilirubin 1 year before and 1 year after the first dose of Oxbryta
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (12)
Rutgers Robert Wood Johnson Medical School Pediatric Clinical Research Center
🇺🇸New Brunswick, New Jersey, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Rutgers Cancer Institute of New Jersey
🇺🇸New Brunswick, New Jersey, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Levine Cancer Institute
🇺🇸Charlotte, North Carolina, United States
University of Texas Health Science Center at Houston
🇺🇸Houston, Texas, United States
University of Connecticut Health
🇺🇸Farmington, Connecticut, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Central Michigan University/Children's Hospital of Michigan
🇺🇸Detroit, Michigan, United States
Parkland Health & Hospital System
🇺🇸Dallas, Texas, United States
Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
Montefiore Medical Center
🇺🇸Bronx, New York, United States