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Recruiting NCT05234567

A Prospective Sub-Study of the Global Hypophosphatasia Registry

— Observational Hypophosphatasia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Asfotase Alfa.
Who it may be relevant to
Registry conditions: Hypophosphatasia. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia

Overview

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.

Interventions

  • Biological 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.

Primary outcome measures

  • Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician [Time frame: Up to 5 years]
  • Occurrence Of Immune-mediated Serious Adverse Events [Time frame: Up to 5 years]

Eligibility 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

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 12 centers
  • Clinical Trial Site — Hartford
  • Clinical Trial Site — Chicago
  • Clinical Trial Site — Boston
  • Clinical Trial Site — Kansas City
  • Clinical Trial Site — Mineola
  • Clinical Trial Site — Cincinnati
  • Clinical Trial Site — Columbus
  • Clinical Trial Site — Pittsburgh
  • … and 4 more centers

Identifiers

NCT: NCT05234567 · ALX-HPP-501s

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗