Gene therapy for classic Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a man between 18 and 50 years old.
- You have classic Fabry disease with very low enzyme activity or a genetic test confirming it.
- You have been on enzyme replacement therapy (ERT) for at least 12 months but still have symptoms like pain or stomach issues.
- Your kidney function is at least 40% of normal (eGFR ≥ 40).
- You weigh 120 kg (264 lbs) or less.
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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