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RECRUITING
NCT05234567

A Prospective Sub-Study of the Global Hypophosphatasia Registry

Sponsor: Alexion Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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.

Official title: 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

Key Details

Gender

All

Age Range

Any - Any

Study Type

OBSERVATIONAL

Enrollment

30

Start Date

2022-08-25

Completion Date

2028-07-18

Last Updated

2026-02-09

Healthy Volunteers

No

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.

Locations (12)

Clinical Trial Site

Hartford, Connecticut, United States

Clinical Trial Site

Chicago, Illinois, United States

Clinical Trial Site

Boston, Massachusetts, United States

Clinical Trial Site

Kansas City, Missouri, United States

Clinical Trial Site

Mineola, New York, United States

Clinical Trial Site

Cincinnati, Ohio, United States

Clinical Trial Site

Columbus, Ohio, United States

Clinical Trial Site

Pittsburgh, Pennsylvania, United States

Clinical Trial Site

Nashville, Tennessee, United States

Clinical Trial Site

Salt Lake City, Utah, United States

Clinical Trial Site

Charlottesville, Virginia, United States

Clinical Trial Site

Madison, Wisconsin, United States