Actively Recruiting
A Prospective Sub-Study of the Global Hypophosphatasia Registry
Led by Alexion Pharmaceuticals, Inc. · Updated on 2026-02-09
30
Participants Needed
12
Research Sites
307 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Prospective Sub-Study of the Global Hypophosphatasia Registry
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Any age or sex with a confirmed diagnosis of pediatric-onset HPP (first sign or symptom before 18 years old)
- Currently receiving asfotase alfa treatment at enrollment or physician has decided to resume or start asfotase alfa treatment within 6 months after enrollment
- Documented low alkaline phosphatase (ALP) activity below normal for age and sex
- Documented ALPL gene mutation, or for infants needing immediate treatment, documentation within 6 months after enrollment
- Ability of participant or parent/legal representative to read and understand informed consent and study questionnaires in local language
- Willingness and ability of participant or parent/legal representative to give signed informed consent, and participant willing to give written assent if required
You will not qualify if you...
- Currently participating in an Alexion-sponsored interventional clinical study. Participants who have completed such studies are eligible.
AI-Screening
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Trial Site Locations
Total: 12 locations
1
Clinical Trial Site
Hartford, Connecticut, United States, 06106
Actively Recruiting
2
Clinical Trial Site
Chicago, Illinois, United States, 60611
Actively Recruiting
3
Clinical Trial Site
Boston, Massachusetts, United States, 02122
Actively Recruiting
4
Clinical Trial Site
Kansas City, Missouri, United States, 64108
Actively Recruiting
5
Clinical Trial Site
Mineola, New York, United States, 11501
Actively Recruiting
6
Clinical Trial Site
Cincinnati, Ohio, United States, 45229
Not Yet Recruiting
7
Clinical Trial Site
Columbus, Ohio, United States, 43203
Actively Recruiting
8
Clinical Trial Site
Pittsburgh, Pennsylvania, United States, 15224
Not Yet Recruiting
9
Clinical Trial Site
Nashville, Tennessee, United States, 37112
Actively Recruiting
10
Clinical Trial Site
Salt Lake City, Utah, United States, 84108
Actively Recruiting
11
Clinical Trial Site
Charlottesville, Virginia, United States, 22903
Actively Recruiting
12
Clinical Trial Site
Madison, Wisconsin, United States, 53792
Actively Recruiting
Research Team
A
Alexion Pharmaceuticals, Inc. (Sponsor)
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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