Registry of Patients With Hypophosphatasia

September 3, 2026 updated by: Alexion Pharmaceuticals, Inc.

An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Detailed Description

The HPP Registry is an observational, prospective, long-term registry designed to collect data on HPP epidemiology, disease history, clinical course, symptoms and burden of disease from patients of all ages who have a diagnosis of HPP.

Evaluation of safety and effectiveness data in patients with HPP who have/are receiving treatment with Asfotase alfa

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
1571

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Herston, Australia
        • Research Site
      • South Brisbane, Australia
        • Research Site
    • Victoria
      • Clayton, Victoria, Australia
        • Research Site
      • Parkville, Victoria, Australia
        • Research Site
  • Austria
      • Linz, Austria
        • Research Site
  • Canada
    • Manitoba
      • Winnipeg, Manitoba, Canada
        • Research Site
    • Ontario
      • Oakville, Ontario, Canada
        • Research Site
      • Ottawa, Ontario, Canada
        • Research Site
      • Toronto, Ontario, Canada, M5G 2C4
        • Research Site
  • Colombia
      • Santander, Colombia, 681003
  • France
      • Lyon, France
        • Research Site
      • Paris, France
        • Research Site
    • Haute Garonne
      • Toulouse, Haute Garonne, France
        • Research Site
    • Nord
      • Lille, Nord, France
        • Research Site
    • Paris
      • Paris, Paris, France
        • Research Site
    • Vienne
      • Poitiers, Vienne, France
        • Research Site
    • Île-de-France Region
      • Le Kremlin-Bicêtre, Île-de-France Region, France
        • Research Site
  • Germany
      • Berlin, Germany
        • Research Site
      • Cologne, Germany
        • Research Site
      • Giessen, Germany
        • Research Site
      • Homburg, Germany
        • Research Site
      • Mainz, Germany
        • Research Site
    • Bavaria
      • Bad Reichenhall, Bavaria, Germany
        • Research Site
      • Würzburg, Bavaria, Germany
        • Research Site
    • North Rhine-Westphalia
      • Bochum, North Rhine-Westphalia, Germany
        • Research Site
      • Düsseldorf, North Rhine-Westphalia, Germany
        • Research Site
    • Saxony
      • Dresden, Saxony, Germany
        • Research Site
  • Italy
      • Florence, Italy
        • Research Site
      • Pisa, Italy
        • Research Site
      • Rome, Italy
        • Research Site
      • San Giovanni Rotondo, Italy
        • Research Site
    • Firenze
      • Sesto Fiorentino, Firenze, Italy
    • Milano
      • Milan, Milano, Italy, 20132
        • Research Site
  • Poland
      • Lodz, Poland
        • Research Site
      • Warsaw, Poland
        • Research Site
  • Saudi Arabia
      • Riyadh, Saudi Arabia
        • Research Site
  • Spain
      • Madrid, Spain
        • Research Site
  • United Kingdom
      • Belfast, United Kingdom
        • Research Site
    • Avon
      • Bristol, Avon, United Kingdom
        • Research Site
    • Greater London
      • London, Greater London, United Kingdom
        • Research Site
    • Leicestershire
      • Leicester, Leicestershire, United Kingdom, LE1 5WW
        • Leicester Royal Infirmary
    • Middlesex
      • Stanmore, Middlesex, United Kingdom
        • Research Site
    • Oxfordshire
      • Oxford, Oxfordshire, United Kingdom
        • Research Site
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom
        • Research Site
    • Tyne & Wear
      • Newcastle upon Tyne, Tyne & Wear, United Kingdom
        • Research Site
    • West Midlands
      • Birmingham, West Midlands, United Kingdom
        • Research Site
  • United States
    • Colorado
      • Centennial, Colorado, United States, 80112
        • Research Site
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Research Site
    • Florida
      • Tampa, Florida, United States, 33606
        • Research Site
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Research Site
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Research Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02111
        • Research Site
      • Boston, Massachusetts, United States, 02115
        • Research Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Research Site
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Research Site
      • St Louis, Missouri, United States, 63110
        • Research Site
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Research Site
    • New York
      • Mineola, New York, United States, 11501
        • Research Site
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Research Site
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Research Site
      • Columbus, Ohio, United States, 43205
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Research Site
    • Tennessee
      • Nashville, Tennessee, United States, 37212
        • Research Site
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Research Site
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Research Site
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

Patients with Hypophosphatasia (HPP)

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Control
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Natural History Information
Time Frame: 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.
1 Year
Burden of Disease/Patient-reported Outcomes
Time Frame: 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)
1 year
Characterize the epidemiology of the HPP population.
Time Frame: 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.
1 year
Long-Term Safety and Effectiveness of Asfotase Alfa
Time Frame: 1 year
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 20, 2015

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2031

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2031

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 26, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 1, 2014

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 3, 2014

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 4, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 3, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ALX-HPP-501

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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