This is an observational study for people with Fabry disease in the US. It looks at the long-term effects of treatments like migalastat HCl and enzyme replacement therapy (ERT) on your health and quality of life. The study aims to understand how these treatments affect your kidney function, specifically your estimated Glomerular Filtration Rate (eGFR), over up to five years. You may be able to join if you are 18 or older, have Fabry disease with specific genetic changes (amenable GLA variants), and have started commercial migalastat treatment within the last two years with an eGFR of 30 mL/min or higher. The study is currently unclear on its recruitment status but plans to enroll 450 participants.

Study design

This is an observational study, meaning participants will be followed over time without being assigned to a specific treatment. It plans to enroll at least 450 participants globally, including those on migalastat, ERT, and those who have never been treated for Fabry disease.

What's involved

Participants will be followed for up to 5 years after they join the study. Your eGFR will be measured at the beginning and annually.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for up to 5 years after enrollment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.