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.
Study Type
OBSERVATIONAL
Enrollment
30
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.
Clinical Trial Site
Hartford, Connecticut, United States
RECRUITINGClinical Trial Site
Chicago, Illinois, United States
RECRUITINGOccurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician
This will be based on clinical and biochemical assessments as well as positive anti-drug antibodies and positive neutralizing antibodies.
Time frame: Up to 5 years
Occurrence Of Immune-mediated Serious Adverse Events
These serious adverse events will include serious hypersensitivity reactions and anaphylaxis.
Time frame: Up to 5 years
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Clinical Trial Site
Boston, Massachusetts, United States
RECRUITINGClinical Trial Site
Kansas City, Missouri, United States
RECRUITINGClinical Trial Site
Mineola, New York, United States
RECRUITINGClinical Trial Site
Cincinnati, Ohio, United States
NOT_YET_RECRUITINGClinical Trial Site
Columbus, Ohio, United States
RECRUITINGClinical Trial Site
Pittsburgh, Pennsylvania, United States
NOT_YET_RECRUITINGClinical Trial Site
Nashville, Tennessee, United States
RECRUITINGClinical Trial Site
Salt Lake City, Utah, United States
RECRUITING...and 2 more locations