Status:
RECRUITING
A Prospective Sub-Study of the Global Hypophosphatasia Registry
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Hypophosphatasia
Eligibility:
All Genders
Brief 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 s...
Eligibility Criteria
Inclusion
- Any age or sex with a confirmed diagnosis of pediatric-onset HPP (that is, first HPP sign or symptom presented at \< 18 years of age).
- Currently receiving asfotase alfa treatment at Enrollment (not treatment-naïve) or the Physician has decided to resume (not treatment-naïve) or start (treatment-naïve) the participant's asfotase alfa treatment within 6 months after Enrollment.
- Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, and a documented ALPL gene mutation (Note: An exception is made for infants with clinical features of HPP plus low ALP who need to start asfotase alfa treatment right away, at the Physician's discretion, but do not yet have a genetic result. In this case, ALPL gene documentation is not required at the time of sub-study enrollment but should be documented within 6 months after Enrollment).
- Participant or participant's parent/legally authorized representative is able to read and/or understand the informed consent and study questionnaires in the local language.
- Participant or participant's parent/legally authorized representative must be willing and able to give signed informed consent for this sub-study, and the participant must be willing to give written informed assent, if appropriate and required by local regulations.
Exclusion
- Currently participating in an Alexion-sponsored interventional clinical study. Participants who have concluded participation in an Alexion-sponsored asfotase alfa clinical study are eligible to enroll in this sub-study.
Key Trial Info
Start Date :
August 25 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
July 18 2028
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT05234567
Start Date
August 25 2022
End Date
July 18 2028
Last Update
August 26 2025
Active Locations (12)
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1
Clinical Trial Site
Hartford, Connecticut, United States, 06106
2
Clinical Trial Site
Chicago, Illinois, United States, 60611
3
Clinical Trial Site
Boston, Massachusetts, United States, 02122
4
Clinical Trial Site
Kansas City, Missouri, United States, 64108