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

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

Detailed Description
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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.

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

Conditions
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Sickle Cell Disease

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


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants who meet all the following criteria will be eligible for enrollment: 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 Male or female participants with documented diagnosis of sickle cell disease (all genotypes) 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: Current participation in an investigation clinical trial or expanded access program, in which the participant may be receiving voxelotor treatment. 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

Ages Eligible for Study
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Minimum Age: 4 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 |
| --- | --- |
| Oxbryta Product Registry<br> | Drug: Oxbryta® (voxelotor) 500mg Tablets<br>* 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.<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, 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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Registry Sickle Cell Disease