A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 216
- 试验地点
- 12
- 主要终点
- Incidence of significant SCD-related clinical events
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- 未提供
研究组 & 干预措施
Retrospective Data Collection
Retrospective Data Collection
干预措施: Oxbryta® (voxelotor) 500-mg Tablets (Drug)
结局指标
主要结局
Incidence of significant SCD-related clinical events
时间窗: 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
时间窗: 1 year before and 1 year after the first dose of Oxbryta
Incidence and severity of serious adverse events (SAEs)
时间窗: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in percent Reticulocytes
时间窗: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in Absolute Reticulocytes
时间窗: 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
时间窗: 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)
时间窗: 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
时间窗: 1 year before and 1 year after the first dose of Oxbryta
Incidence and severity of adverse events (AEs) of interest
时间窗: 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)
时间窗: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in Bilirubin
时间窗: 1 year before and 1 year after the first dose of Oxbryta
次要结局
未报告次要终点