Safety and effectiveness of an experimental infusion for Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 years or older
- You must have a confirmed genetic diagnosis of Fabry disease and at least one symptom
- You may or may not have received enzyme replacement therapy (ERT) before
- You must have kidney or heart involvement from Fabry disease
- Your liver and blood test results must be within certain limits (no severe liver problems)
- You must not have serious heart disease, active infections, autoimmune diseases, cancer (except certain skin or early-stage cancers), or a history of certain mental conditions
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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