In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.
Eligibility
Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Male and female participants, of any age, with a confirmed diagnosis of HPP.
* Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
* Participant or legal representative is able to read and/or understand the informed consent and study questionnaires in the local language.
* Signed informed consent and medical records release by the patient or legal representative. Patient or patient's parent/legal representative must be willing and able to give written informed consent, and the patient must be willing to give written informed assent, if appropriate and required by local regulations.
Exclusion Criteria:
* Currently participating in an Alexion-sponsored clinical trial. Enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.
Primary outcome measure(s)
Natural History Information — 1 Year To collect information on the natural history of HPP from patients of all ages, including pediatric patients and adults with HPP, regardless of age at onset.
Burden of Disease/Patient-reported Outcomes — 1 year Age-appropriate PRO data will be collected using instruments to asses below: Pain, Motor Capacity, Functional Status/Disability, including Activities of Daily Living (ADL), Quality of Life (QoL)
Characterize the epidemiology of the HPP population. — 1 year To characterize the epidemiology of the HPP population. Inclusion of all classifications of HPP is planned: pediatric-onset (perinatal-, infantile-, and juvenile-onset), adult-onset, benign perinatal, and odontohypophosphatasia.
Long-Term Safety and Effectiveness of Asfotase Alfa — 1 year To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.
Trial sites (66)
Facility
City
Region
Status
Research Site
Centennial
Colorado
Research Site
Hartford
Connecticut
Research Site
Tampa
Florida
Research Site
Decatur
Georgia
Research Site
Baltimore
Maryland
Research Site
Boston
Massachusetts
Research Site
Boston
Massachusetts
Research Site
Minneapolis
Minnesota
Research Site
Kansas City
Missouri
Research Site
St Louis
Missouri
Research Site
Hackensack
New Jersey
Research Site
Mineola
New York
Research Site
Durham
North Carolina
Research Site
Cincinnati
Ohio
Research Site
Columbus
Ohio
Research Site
Pittsburgh
Pennsylvania
Research Site
Nashville
Tennessee
Research Site
Salt Lake City
Utah
Research Site
Charlottesville
Virginia
Research Site
Madison
Wisconsin
Research Site
Clayton
Victoria
Research Site
Parkville
Victoria
Research Site
Herston
Australia
Research Site
South Brisbane
Australia
Research Site
Linz
Austria
Research Site
Winnipeg
Manitoba
Research Site
Oakville
Ontario
Research Site
Ottawa
Ontario
Research Site
Toronto
Ontario
—
Santander
Colombia
Research Site
Toulouse
Haute Garonne
Research Site
Lille
Nord
Research Site
Paris
Paris
Research Site
Poitiers
Vienne
Research Site
Lyon
France
Research Site
Paris
France
Research Site
Le Kremlin-Bicêtre
Île-de-France Region
Research Site
Bad Reichenhall
Bavaria
Research Site
Würzburg
Bavaria
Research Site
Bochum
North Rhine-Westphalia
+ 26 more sites — see the full list on the official registry below.
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.
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