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A Prospective Sub-Study of the Global Hypophosphatasia Registry

NCT05234567 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 30

Last updated 2026-02-09

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-25
Primary Completion
2028-07-18
Completion
2028-07-18
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05234567 on ClinicalTrials.gov