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A Prospective Sub-Study of the Global Hypophosphatasia Registry
Sponsor: Alexion Pharmaceuticals, Inc.
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
Conditions
Interventions
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