PRX-102 for children with Fabry disease
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This study tests a new enzyme replacement therapy called PRX-102 for children with Fabry disease. It aims to see if the drug is safe and helps manage symptoms like pain and eye or skin changes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be a child age 2 to 17 years old.
- You must have a confirmed diagnosis of Fabry disease.
- You must have at least one Fabry symptom like nerve pain, cornea verticillata (a cloudy swirl in the eye), or angiokeratomas (small red or purple spots on skin).
- You must have a history of Fabry pain (crises or chronic pain).
- Your doctor must think you need enzyme replacement therapy (ERT) for Fabry disease.
- You cannot have severe kidney problems, a history of severe allergic reaction to other Fabry treatments, or certain other serious health conditions.
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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