Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Number and proportion of patients at high-risk for FD in the CCAD EMR database
研究概览
简要总结
Primary objective:
To estimate the prevalence of patients who are at high-risk for Fabry Disease (FD) in the Cleveland Clinic, Abu Dhabi (CCAD) United Arab Emirates (EMR) database from May 2016 to May 2022, according to the predictive algorithm (i.e., feasibility assessment eligibility criteria)
Secondary objectives:
- To estimate the prevalence of FD among patients at high-risk for FD (i.e., among enrolled patients)
- To characterize the patient profile, overall and in Cohorts 1 and 2
- To describe the most common characteristics among positive FD patients and negative FD patients
详细描述
Patients at high-risk for FD will be consecutively enrolled over approximately 6 months
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who were identified as high-risk for FD via the predictive algorithm per the feasibility assessment
- •Patients who have signed an informed consent form
- •Cohort 1 (chronic kidney disease)
- •Adult male patient ≤60 years old or adult female patient of any age
- •Having chronic kidney disease
- •Having proteinuria
- •Having one or more of the following conditions:
- •Neuralgia and neuritis, unspecified
- •Disturbances in skin sensation
- •Anhidrosis, and
- •Hypertrophic cardiomyopathy.
- •Cohort 2 (hypertrophic cardiomyopathy)
- •Adult male patient ≤50 years old or adult female patient of any age
- •Having hypertrophic cardiomyopathy
- •Having one or more of the following conditions:
- •transient cerebral ischemic attack,
- •chronic kidney disease.
排除标准
- •Patients with an established diagnosis of FD.
- •Pregnant subject at the time of the study.
- •The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
结局指标
主要结局
Number and proportion of patients at high-risk for FD in the CCAD EMR database
时间窗: Up to 6 months
The prevalence of patients at high-risk for FD in the CCAD EMR database, according to the predictive algorithm, from May 2016 to May 2022, will be estimated. The percentage (and 95% confidence interval \[CI\]) of patients at high-risk for FD will be calculated, overall and in Cohorts 1 and 2.
次要结局
- Number and proportion of patients with FD (overall and among patients in Cohorts 1 and 2)(Up to 6 months)
- To describe the following patient characteristics, among positive FD patients and negative FD cases:(Up to 6 months)
- To describe the following patient characteristics, overall and in Cohorts 1 and 2:(Up to 6 months)