A Prospective Sub-Study of the Global Hypophosphatasia Registry
Condition(s) studied
Investigational drug(s) / intervention(s)
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.
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.
Eligibility
Primary outcome measure(s)
- Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician — Up to 5 years
This will be based on clinical and biochemical assessments as well as positive anti-drug antibodies and positive neutralizing antibodies. - Occurrence Of Immune-mediated Serious Adverse Events — Up to 5 years
These serious adverse events will include serious hypersensitivity reactions and anaphylaxis.
Trial sites (12)
| Facility | City | Region | Status |
|---|---|---|---|
| Clinical Trial Site | Hartford | Connecticut | Recruiting |
| Clinical Trial Site | Chicago | Illinois | Recruiting |
| Clinical Trial Site | Boston | Massachusetts | Recruiting |
| Clinical Trial Site | Kansas City | Missouri | Recruiting |
| Clinical Trial Site | Mineola | New York | Recruiting |
| Clinical Trial Site | Cincinnati | Ohio | Not Yet Recruiting |
| Clinical Trial Site | Columbus | Ohio | Recruiting |
| Clinical Trial Site | Pittsburgh | Pennsylvania | Not Yet Recruiting |
| Clinical Trial Site | Nashville | Tennessee | Recruiting |
| Clinical Trial Site | Salt Lake City | Utah | Recruiting |
| Clinical Trial Site | Charlottesville | Virginia | Recruiting |
| Clinical Trial Site | Madison | Wisconsin | Recruiting |
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05234567 on ClinicalTrials.gov ↗ ← All trials in the USA