Study of Fabry disease treatment in Chinese children and adults
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 7 years old or older.
- You have a confirmed diagnosis of Fabry disease.
- You have either never had enzyme replacement therapy (ERT) or received it within the past 12 weeks.
- You are not currently in another Fabry disease clinical trial.
- You do not have severe heart failure, a recent heart attack, a serious stroke, or a certain type of heart block (unless you have a pacemaker or defibrillator).
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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This trial tests whether agalsidase alfa can reduce heart inflammation in people with Fabry disease who have heart problems. You may qualify if you have not had this treatment in the past year.
This study checks whether certain heart-related blood tests and heart scans can help monitor people with Fabry disease. It may be useful for both people who have never received enzyme replacement therapy and those who have.
This trial tests if enzyme replacement therapy works well and is safe for Fabry disease. It is for people who are already prescribed this treatment.
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 study tests a new gene therapy for children aged 7 to 17 with Fabry disease. The treatment aims to fix the faulty gene that causes the condition, which may help reduce symptoms and prevent organ damage.
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