Study of a new drug for Fabry disease
Part of Genetic & congenital, Kidney & urinary 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.
Similar studies
Other trials that look related to this one.
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This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.
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.
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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 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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