Registry of Patients With Hypophosphatasia

July 11, 2023 updated by: Alexion

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

Enrolling by invitation

Conditions

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

Observational

Enrollment (Estimated)

900

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
      • South Brisbane, Australia
    • New South Wales
      • Westmead, New South Wales, Australia
    • Victoria
      • Clayton, Victoria, Australia
      • Parkville, Victoria, Australia
  • Canada
    • Manitoba
      • Winnipeg, Manitoba, Canada
  • France
      • Lyon, France
    • Haute Garonne
      • Toulouse, Haute Garonne, France
    • Paris
      • Paris cedex 14, Paris, France
    • Île-de-France
      • Le Kremlin-Bicêtre, Île-de-France, France
  • Germany
      • Berlin, Germany
      • Giessen, Germany
      • Hamburg, Germany
      • Homburg, Germany
      • Koeln, Germany
      • Mainz, Germany
      • Muenchen, Germany
      • Müllheim, Germany
      • Rostock, Germany
    • Bayern
      • Bad Reichenhall, Bayern, Germany
      • Würzburg, Bayern, Germany
    • Nordrhein Westfalen
      • Bochum, Nordrhein Westfalen, Germany
      • Düsseldorf, Nordrhein Westfalen, Germany
    • Sachsen
      • Dresden, Sachsen, Germany
  • Italy
      • Firenze, Italy
      • Pisa, Italy
      • Rome, Italy
      • San Giovanni Rotondo, Italy
      • Verona, Italy
  • Poland
      • Warsaw, Poland
  • Russian Federation
      • Moscow, Russian Federation
      • Saint Petersburg, Russian Federation
  • Saudi Arabia
      • Riyadh, Saudi Arabia
  • Spain
      • Barcelona, Spain
      • Madrid, Spain
  • United Kingdom
      • Belfast, United Kingdom
      • Birmingham, United Kingdom
      • Dundee, United Kingdom
      • Leicester, United Kingdom
      • London, United Kingdom
      • Newcastle upon Tyne, United Kingdom
      • Newport, United Kingdom
      • Norwich, United Kingdom
      • Oxford, United Kingdom
      • Sheffield, United Kingdom
      • Stanmore, United Kingdom
    • Avon
      • Bristol, Avon, United Kingdom
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
    • Colorado
      • Centennial, Colorado, United States, 80112
      • Golden, Colorado, United States, 80401
    • Connecticut
      • Hartford, Connecticut, United States, 06106
    • Florida
      • Tampa, Florida, United States, 33606
    • Georgia
      • Decatur, Georgia, United States, 30033
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Maryland
      • Baltimore, Maryland, United States, 21287
    • Massachusetts
      • Boston, Massachusetts, United States, 02111
    • Missouri
      • Kansas City, Missouri, United States, 64108
      • Saint Louis, Missouri, United States, 63110
    • New York
      • Mineola, New York, United States, 11501
      • Rochester, New York, United States, 14642
      • Syracuse, New York, United States, 13214
    • North Carolina
      • Durham, North Carolina, United States, 27710
    • Ohio
      • Cincinnati, Ohio, United States, 45229
      • Columbus, Ohio, United States, 43205
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
    • Tennessee
      • Nashville, Tennessee, United States, 37212
    • Utah
      • Salt Lake City, Utah, United States, 84112
    • Wisconsin
      • Madison, Wisconsin, United States, 53792

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

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

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

Alexion

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)

December 31, 2014

Primary Completion (Estimated)

December 31, 2024

Study Completion (Estimated)

August 28, 2030

Study Registration Dates

First Submitted

November 26, 2014

First Submitted That Met QC Criteria

December 1, 2014

First Posted (Estimated)

December 3, 2014

Study Record Updates

Last Update Posted (Actual)

July 12, 2023

Last Update Submitted That Met QC Criteria

July 11, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID 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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