Clinical trial NCT04930328: Retrospective Real World Oxbryta® Data Collection and Analysis Study
Official title: A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Keywords: Sickle Cell Disease Registry
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-06-18
Enrollment: 216
Sex: ALL
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
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: 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.

Eligibility criteria: Inclusion Criteria:

Patients who meet all the following criteria will be eligible for inclusion in this study:

1. 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
2. Male or female patients with documented diagnosis of SCD (all genotypes)
3. Have been treated with Oxbryta for at least 2 weeks, according to the Oxbryta USPI

Exclusion Criteria:

-

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: Farmington
country: United States
facility: University of Connecticut Health
state: Connecticut
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Detroit
country: United States
facility: Central Michigan University/Children's Hospital of Michigan
state: Michigan
city: New Brunswick
country: United States
facility: Rutgers Robert Wood Johnson Medical School Pediatric Clinical Research Center
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute of New Jersey
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 Medical Center
state: North Carolina
city: Dallas
country: United States
facility: Parkland Health & Hospital System
state: Texas
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: University of Texas Health Science Center at Houston
state: Texas
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: