Long-term study of Fabry disease treatments in the US
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This study looks at how well available Fabry disease treatments (like migalastat or ERT) work over time in real life. It may help doctors understand who benefits most from these medicines.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 or older and have a confirmed diagnosis of Fabry disease.
- Your kidney function (eGFR) must be 30 or higher at the time you join.
- If you take migalastat (Galafold), you must have a genetic variant that is 'amenable' to the drug.
- If you take enzyme replacement therapy (ERT), you must have started it within the last 2 years.
- You must show signs of Fabry disease getting worse, like kidney decline or protein in urine (or be a male with classic Fabry disease).
- You cannot be in any other clinical trial for a study drug or device right now.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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