Retrospective Real World Oxbryta® Data Collection and Analysis Study

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: Oxbryta® (voxelotor) 500-mg Tablets


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: -

Ages Eligible for Study
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Minimum Age: 0 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Retrospective Data Collection<br>Retrospective Data Collection | Drug: Oxbryta® (voxelotor) 500-mg Tablets<br>* Patients will have received treatment with Oxbryta as prescribed by their physician at the approved dose per local prescribing information, as part of their usual care.<br>* Other names: Oxbryta®;|

What is the study measuring?
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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, and 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Sickle Cell Disease Registry