Ireland
--:--IST
Recruiting Observational

A Prospective Sub-Study of the Global Hypophosphatasia Registry

NCT05234567 · tracked via the Priya Life Science USA tracker
Phase
Observational
Started
2022-08-25
Last updated
2026-02-09

Condition(s) studied

Hypophosphatasia

Investigational drug(s) / intervention(s)

Asfotase Alfa →

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

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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: * 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.

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)

FacilityCityRegionStatus
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
Official registry record

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