Enzyme therapy study for Fabry disease patients
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with Fabry disease
- You are between 8 and 65 years old
- You have been prescribed a course of enzyme replacement therapy (Fabagal, Fabrazyme, Replagal)
- You are not on dialysis or have not had a kidney transplant
- You are not pregnant or breastfeeding
- You and/or your legal representative agree to join the study and share your data
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 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 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 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 studies Replagal (enzyme replacement therapy) for people with Fabry disease who have not used it before. It aims to see how the treatment works and how safely it can be used in children and adults.
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.
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.
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.