A Prospective Sub-Study of the Global Hypophosphatasia Registry
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- 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 CRITERIA
Exclusion Criteria:
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.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Hypophosphatasia
Participants needed
40
Est. Completion Date
Jun 30, 2028
Treatment type
Observational
Sponsor
Alexion Pharmaceuticals, Inc.
ClinicalTrials.gov identifier
NCT05234567
Study number
ALX-HPP-501s
Understanding Clinical Trials
Get answers to your questions about clinical trials.What is a clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?