Study drug PRX-102 for Fabry disease in Japanese patients
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests whether PRX-102 is safe and effective for treating Fabry disease in Japanese participants. It also measures how the drug moves through the body and affects body systems, to see if it helps your symptoms and organ function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (and your parents and all 4 grandparents) must be of Japanese descent, and you must be born in Japan
- You must have a confirmed Fabry disease diagnosis, plus at least one typical sign (nerve pain, cornea deposits, or clustered skin blood-vessel spots)
- Your kidney function must be at least moderate (eGFR 40 or higher at screening)
- Your doctor must feel you need enzyme replacement therapy (ERT) for Fabry disease
- If you are in the trial for kidney-worsening type (Cohort A), you must be an adult and show worsening kidney function in recent labs
- You cannot have had certain Fabry medicines/related treatments very recently before the trial starts
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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