Study of a new drug for Fabry disease
Part of Genetic & congenital clinical trials.
This study tests a new investigational drug called HM15421/GC1134A for people with Fabry disease. It aims to see if the drug is safe and tolerable, and to learn how well it works. You may be eligible if you have Fabry disease with symptoms, have not had treatment for at least 6 months, and your kidneys are functioning well.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 years or older.
- You have been diagnosed with Fabry disease and have symptoms.
- You have not taken any Fabry disease treatment (including experimental drugs) in the past 6 months, and you test negative for antibodies against the study drug.
- Your kidneys work well (eGFR at least 60) and you have a specific blood marker (lyso-Gb3) that is higher than normal.
- You do not have serious kidney disease, heart failure, or a history of stroke, and you are not pregnant or breastfeeding.
- You are willing to use effective contraception (if applicable) and follow study rules.
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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