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.
Similar studies
Other trials that look related to this one.
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 a new medicine called EXG110, given through a vein, for people with Fabry disease. It aims to see if the medicine is safe and helps with symptoms related to the heart or kidneys.
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 tests a new enzyme replacement therapy called PRX-102 for children with Fabry disease. It aims to see if the drug is safe and helps manage symptoms like pain and eye or skin changes.
This trial is testing a new enzyme replacement therapy called Fabagal for people with Fabry disease who have not had prior treatment. It aims to see if it helps with kidney, heart, or brain problems caused by the disease.
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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