An observational study in Hypophosphatasia, sponsored by Alexion Pharmaceuticals, Inc.. Recruiting at 12 sites in United States. Per ClinicalTrials.gov, last updated 2026-02-09.
Sponsored by Alexion Pharmaceuticals, Inc. · Observational
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.
45 studies on the registry are indexed under Hypophosphatasia; 10 are open to participants now.
This study's planned enrollment of 30 is below the median of 58 across 22 observational studies indexed under Hypophosphatasia.
Browse Hypophosphatasia studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 249 studies on the registry; 25 are open to participants now.
Of its 98 completed or terminated interventional studies of FDA-regulated products, 70 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients with HPP
Exclusion Criteria:
Each participant will be followed for a minimum of 5 years or, if applicable, until early withdrawal. Biochemical, clinical, imaging (if clinically indicated), and functional/quality of life outcomes relevant to HPP will be assessed.
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.
Also known as: Strensiq
Occurrence 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
Plan to share: Yes — Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Supporting information: Study protocol, Sap, Csr
No publications or documents are linked to this record.
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Alexion Pharmaceuticals, Inc.