Hypophosphatasia (HPP) Clinical Trials

• Age ≥4 to \<18 years, or ≥18 years at the time of enrollment;

• Signed ICF for patients ≥18 years, or legal representatives (parents) of patients aged ≥4 to \<18 years;

• Written informed assent (for patients aged ≥14 to \<18 years only);

• No history of HPP treatment with enzyme-replacement therapy;

• Diagnosis of HPP confirmed by:

‣ reduced alkaline phosphatase (ALP) activity relative to age- and sex-specific reference ranges, confirmed by at least two separate measurements, AND

⁃ the identification of a monoallelic pathogenic, likely pathogenic, or variant of uncertain significance in the ALPL gene on genetic testing.