A Prospective, Multi-center, Observational Study to Evaluate the Clinical Outcomes of Agalsidase Alfa Enzyme Replacement Therapy Among Chinese Patients With Fabry Disease in Real-world Clinical Practice
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 200
- 试验地点
- 18
- 主要终点
- Annualized Rate of Change in Left Ventricular Mass Index (LVMI)
研究概览
简要总结
Fabry Disease is a rare blood disorder that some people are born with. People with Fabry disease have low levels of an enzyme called alpha-galactosidase A. This enzyme helps to cut down fat-like substances. Without alpha-galactosidase A, large forms of these substances build up and clot in blood vessels. Over time, this can affect vital organs (especially the heart, kidneys, and brain) causing serious health problems with advancing age. Agalsidase alfa (Replagal®) is a human enzyme made in the laboratory and may provide higher levels of alpha-galactosidase A. Replagal® works the same way as natural alpha-galactosidase A does.
The main aim of this study is to learn more about the treatment with Replagal® in Chinese children and adults with Fabry disease. The study aims to assess the heart and kidney function in people with Fabry disease who are routinely treated with Replagal®. Other aims are to learn about the change in heart and kidney function, impact on quality of life, how the treatment with Replagal® works for people with Fabry Disease, and how safe the treatment with Replagal® is in routine real-world settings.
Participants will receive with Replagal® per the routine treatment settings in China. No study-specific visits to the clinical are scheduled.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Annualized Rate of Change in Left Ventricular Mass Index (LVMI)
时间窗: Up to 18 months
The annualized rate of change in LVMI will be estimated using linear mixed effects model.
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR)
时间窗: Up to 18 months
次要结局
- Annualized Rate of Change in Left Ventricular Posterior Wall Dimensions (LVPWD)(Up to 18 months)
- Annualized Rate of Change in Ejection Fraction (EF)(Up to 18 months)
- Change From Baseline Over Time in LVMI(Baseline, up to 18 months)
- Change From Baseline Over Time in LVPWD(Baseline, up to 18 months)
- Change From Baseline Over Time in EF(Baseline, up to 18 months)
- Annualized Rate of Change in Urinary Albumin to Creatinine Ratio (UACR)(Up to 18 months)
- Annualized Rate of Change in Urine Protein Creatine Ratio (UPCR)(Up to 18 months)
- Change From Baseline Over Time in eGFR(Baseline, up to 18 months)
- Change From Baseline Over Time in UACR(Baseline, up to 18 months)
- Change From Baseline Over Time in UPCR(Baseline, up to 18 months)
- Change From Baseline Over Time in 24-hour (h) Urine Protein(Baseline, up to 18 months)
- Change From Baseline Over Time in 36-Item Short Form Health Survey (SF-36)(Baseline, up to 18 months)
- Change From Baseline Over Time in Mainz Severity Score Index (MSSI)(Baseline, up to 18 months)
- Number of Participants With Clinically Significant Adverse Events (AEs) With Agalsidase Alfa Treatment(Up to 18 months)