Testing a new IV treatment for Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 years or older and have Fabry disease confirmed by genetic testing.
- You have at least one Fabry symptom and problems with your heart or kidneys.
- You may or may not have had enzyme replacement therapy before.
- You do not have serious liver issues or active infections like hepatitis B or C.
- You have not had gene therapy or another experimental treatment in the last 3 months.
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
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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 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 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 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 Phase 1/2 study tests how safe and potentially effective 4D-310 is for adults with Fabry disease who have heart involvement. It may help people who can’t tolerate standard treatments like enzyme replacement therapy (ERT) or migalastat, or who still have disease progression.
This study tests an oral medication called AL01211 for men who have classic Fabry disease but have never been treated before. The goal is to see if it is safe and if it can help reduce the build-up of fatty substances in the body.
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