NCT05234567

A Prospective Sub-Study of the Global Hypophosphatasia Registry

A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 30 (estimated)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

Conditions

• Hypophosphatasia

Interventions

Type	Name	Description
BIOLOGICAL	Asfotase Alfa	All participants will receive asfotase alfa subcutaneously per standard of care. Unless otherwise specified per the Physician's standard of care, participants aged \< 2 years are recommended for a clinic visit approximately every 3 months after Enrollment until 2 years of age, after which they should have a clinic visit approximately every 6 months. Participants should be followed for 5 years, as possible.

Timeline

• Start date: 2022-08-25
• Primary completion: 2028-07-18
• Completion: 2028-07-18
• First posted: 2022-02-10
• Last updated: 2026-02-09

Locations

12 sites across 1 country: United States

Regulatory

• FDA-regulated drug study