The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Asfotase alfa is administered subcutaneously.
Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1
Time frame: Baseline, Week 36
Change From Baseline To Week 36 In Plasma Concentrations Of PPi In Group 2
Time frame: Baseline, Week 36
Change From Baseline To Week 36 In The 36-item Short-Form Survey (SF-36) Physical Component Summary (PCS) Score In Groups 1 And 2
Time frame: Baseline, Week 36
Change From Baseline To Week 36 In The Repeated Chair Stand Test (A Component Of The Short Physical Performance Battery [SPPB]) In Groups 1 And 2
Time frame: Baseline, Week 36
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