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.
Similar studies
Other trials that look related to this one.
This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.
This study is for people with Fabry disease who are taking or planning to take the medication Elfabrio (pegunigalsidase alfa). It will look at how safe and effective the treatment is in real-world use.
This trial tests a new gene therapy called AMT-191 for men with classic Fabry disease who still have symptoms despite standard enzyme replacement therapy. It aims to see if the treatment is safe and can improve symptoms.
This study observes how well the medication pegunigalsidase alfa works over a long period in people with Fabry disease. It helps doctors learn more about managing the condition in everyday life.
This study tests an enzyme replacement therapy (agalsidase alfa) for Fabry disease in Chinese children and adults. It's an observational study that follows patients who receive the treatment as part of their regular care.
This study follows pregnant and breastfeeding women with Fabry disease (and their infants) to better understand what happens during pregnancy and early life. You may or may not be taking migalastat, and the goal is to gather real-world information that could help future care.
Hear when a new Fabry Disease trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.