Observing the Effects of Agalsidase Alfa Enzyme Replacement Therapy in Chinese Children and Adults With Fabry Disease in Everyday Clinical Use

What this Trial Is About

Fabry Disease is a rare inherited blood disorder caused by low levels of an enzyme called alpha-galactosidase A. This enzyme normally breaks down fat-like substances, but without enough of it, these substances can build up in blood vessels and harm vital organs such as the heart, kidneys, and brain over time. This study aims to learn more about the treatment effects of agalsidase alfa Replagal4, an enzyme replacement therapy, in Chinese children and adults with Fabry disease by observing its impact on heart and kidney function, quality of life, and safety in routine clinical use. Participants in this study will receive enzyme replacement therapy with agalsidase alfa as part of their usual clinical care in China. This is a non-interventional, observational study where no additional study-specific visits are scheduled. The treatment follows routine practice settings, and researchers will monitor participants receiving this therapy over time. During the study lasting up to 18 months, participants will be observed for changes in heart function using measures like left ventricular mass index and ejection fraction, and kidney function using estimated glomerular filtration rate and urine analyses. Quality of life assessments and monitoring of adverse events will also be conducted. The study collects data from regular clinical visits without extra procedures, aiming to understand how agalsidase alfa works and its safety in real-world treatment of Fabry disease.

A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease