Gene therapy study for children with Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 7 and 17 years old.
- You must have a confirmed Fabry disease diagnosis through genetic testing.
- You must have at least one symptom of Fabry disease, like pain, rash, or kidney issues.
- Your kidney function needs to be at a certain level.
- You cannot have certain infections like hepatitis, HIV, or syphilis.
- You must not have had other gene therapy or experimental drugs recently.
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 is testing a new treatment called EXG110 for people with Fabry disease. It's designed to see if it's safe and if it can help. You may be able to join if you have a confirmed diagnosis and at least one symptom of Fabry disease.
This trial tests a one-time gene therapy called AMT-191 for men with classic Fabry disease who are still having symptoms despite enzyme replacement therapy. The goal is to see if it can reduce pain and stomach problems and improve overall health.
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 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 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 tests a new medicine given through an IV that may help treat Fabry disease. It focuses on people with kidney or heart problems caused by the disease, and may be an option if standard enzyme replacement therapy hasn't worked or isn't being used.
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.