Testing a new gene therapy for Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 7 years old or older.
- You have been diagnosed with Fabry disease through genetic testing and have at least one symptom of the disease.
- You may have had enzyme replacement therapy before or not.
- If you are an adult, you have problems with your kidneys or heart due to Fabry disease.
- If you could have children, you agree to use birth control and not donate sperm for up to a year after treatment.
- You are willing to have tests and give samples as part of the study.
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 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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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.
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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.
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