Study of children with HPP on (or starting) asfotase alfa
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This study follows children with hypophosphatasia (HPP) to better understand how they do while receiving asfotase alfa. It may help improve care for families living with pediatric-onset HPP.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with pediatric-onset HPP, meaning symptoms started before age 18
- You are already taking asfotase alfa, or your doctor plans to start/resume it within 6 months
- Your lab test shows low alkaline phosphatase (ALP) for your age/sex
- You have an ALPL gene change documented (unless your baby must start treatment right away; then it can be documented within 6 months)
- You (or a parent/guardian) can read and understand the study materials and consent forms in your local language
- You are not currently in another Alexion-sponsored interventional trial (past Alexion asfotase alfa studies can be eligible)
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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