Gene therapy for classic Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests a one-time gene therapy called AMT-191 for men with classic Fabry disease who are still having symptoms despite enzyme replacement therapy. The goal is to see if it can reduce pain and stomach problems and improve overall health.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be a man aged 18 to 65 years
- Have a confirmed diagnosis of classic Fabry disease with very low or absent enzyme activity
- Have kidney function that is not too low (eGFR of 40 or higher)
- Have been on enzyme replacement therapy for at least 12 months with persistent symptoms such as pain or stomach issues
- Weigh between 45 and 120 kilograms (about 99 to 264 pounds)
- Agree to use a condom with female partners who can become pregnant
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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