Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
Status:
Completed
Trial end date:
2016-09-01
Target enrollment:
Participant gender:
Summary
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by
gene mutations or changes. These gene mutations cause low levels of an enzyme needed to
harden bone. The purpose of this study was to test the safety and efficacy of a study drug
called asfotase alfa (human recombinant tissue non-specific alkaline phosphate fusion
protein) to see what effects it has on patients 5 years of age or less with HPP.