Registry of Patients With Hypophosphatasia
Launched by ALEXION PHARMACEUTICALS, INC. · Dec 1, 2014
Trial Information
Current as of September 12, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
This is an observational, long-term registry called the Hypophosphatasia (HPP) Registry. It follows people of all ages who have HPP to learn how the disease behaves in real life, what symptoms people experience, and how it affects daily life. The study also collects information on safety and how well the treatment asfotase alfa works over time for those who are receiving it. It’s not a randomized trial; researchers simply observe and record information over years, with a focus on one-year snapshots and long-term experience.
Who can join? Anyone with a confirmed diagnosis of HPP, of any age or sex, who has low alkaline phosphatase (ALP) activity for their age/sex or a known ALPL gene mutation, and who can understand and sign the consent forms. Participants (or their guardians) must be willing to provide consent. If you’re currently in another Alexion-sponsored trial, you can still join the registry after that trial ends. The study has sites around the world—across the United States, Europe, Canada, Australia, and other countries—with enrollment estimated up to about 900 people and planned follow-up lasting several years, including at least one year of data collection on natural history and patient-reported outcomes, plus long-term safety and effectiveness data for asfotase alfa. Enrollment is ongoing by invitation, with study completion anticipated in 2031.
Gender
ALL
Eligibility criteria
- 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.
About Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc. is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapies for patients with rare and complex diseases. With a strong focus on addressing unmet medical needs, Alexion leverages advanced science and technology to create transformative treatments that improve the quality of life for patients suffering from conditions such as complement-mediated disorders, neurology, hematology, and metabolic diseases. Committed to patient-centric approaches, Alexion collaborates with healthcare professionals, patients, and advocacy groups to drive clinical research and foster advancements in rare disease treatment.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Decatur, Georgia, United States
Rochester, New York, United States
Baltimore, Maryland, United States
Warsaw, Poland
Barcelona, Spain
Madrid, Spain
London, United Kingdom
Berlin, Germany
Hamburg, Germany
Pisa, Italy
Moscow, Russian Federation
Chicago, Illinois, United States
Boston, Massachusetts, United States
Bristol, Avon, United Kingdom
Parkville, Victoria, Australia
Winnipeg, Manitoba, Canada
Tampa, Florida, United States
Madison, Wisconsin, United States
Belfast, United Kingdom
Hartford, Connecticut, United States
Mineola, New York, United States
Saint Louis, Missouri, United States
Durham, North Carolina, United States
Chicago, Illinois, United States
Rochester, New York, United States
Durham, North Carolina, United States
Nashville, Tennessee, United States
Newcastle Upon Tyne, United Kingdom
Cincinnati, Ohio, United States
Lyon, France
Berlin, Germany
London, United Kingdom
Birmingham, United Kingdom
Dundee, United Kingdom
Hamburg, Germany
Mainz, Germany
Linz, Austria
Warszawa, Poland
Moscow, Russian Federation
Barcelona, Spain
Madrid, Spain
Winnipeg, Manitoba, Canada
Ottawa, Ontario, Canada
Toronto, Ontario, Canada
Pittsburgh, Pennsylvania, United States
Lyon, France
Belfast, United Kingdom
Dundee, United Kingdom
Giessen, Germany
Saint Petersburg, Russian Federation
Leicester, United Kingdom
Sheffield, United Kingdom
Birmingham, United Kingdom
Rostock, Germany
Mainz, Germany
Firenze, Italy
Westmead, New South Wales, Australia
Rome, Italy
Homburg, Germany
Firenze, Italy
Pisa, Italy
Oxford, United Kingdom
Salt Lake City, Utah, United States
Saint Petersburg, Russian Federation
Leicester, United Kingdom
Sheffield, United Kingdom
Westmead, New South Wales, Australia
Kansas City, Missouri, United States
Newcastle Upon Tyne, United Kingdom
Warsaw, Poland
Baltimore, Maryland, United States
Oxford, United Kingdom
Giessen, Germany
Verona, Italy
Newport, United Kingdom
Dresden, Sachsen, Germany
Newport, United Kingdom
Herston, Australia
South Brisbane, Australia
Madison, Wisconsin, United States
Riyadh, Saudi Arabia
Rome, Italy
San Giovanni Rotondo, Italy
Oakville, Ontario, Canada
Verona, Italy
Parkville, Victoria, Australia
Birmingham, West Midlands, United Kingdom
Herston, Australia
Salt Lake City, Utah, United States
Newcastle Upon Tyne, United Kingdom
Cincinnati, Ohio, United States
Norwich, United Kingdom
Rostock, Germany
Columbus, Ohio, United States
Homburg, Germany
Muenchen, Germany
Kansas City, Missouri, United States
Paris, France
Muenchen, Germany
Wuerzburg, Germany
Charlottesville, Virginia, United States
Saint Louis, Missouri, United States
Hackensack, New Jersey, United States
Pittsburgh, Pennsylvania, United States
Berlin, Germany
Norwich, United Kingdom
Centennial, Colorado, United States
Little Rock, Arkansas, United States
Koeln, Germany
South Brisbane, Australia
Clayton, Victoria, Australia
Centennial, Colorado, United States
Riyadh, Saudi Arabia
Boston, Massachusetts, United States
Mineola, New York, United States
Columbus, Ohio, United States
Little Rock, Arkansas, United States
Tampa, Florida, United States
Stanmore, United Kingdom
Nashville, Tennessee, United States
Clayton, Victoria, Australia
Decatur, Georgia, United States
Stanmore, United Kingdom
Koeln, Germany
Hartford, Connecticut, United States
Dresden, Sachsen, Germany
Würzburg, Bayern, Germany
Golden, Colorado, United States
Lodz, Poland
Herston, Queensland, Australia
Barcelona, Cataluña, Spain
Minneapolis, Minnesota, United States
London, Greater London, United Kingdom
Leicester, Leicestershire, United Kingdom
San Giovanni Rotondo, Italy
Golden, Colorado, United States
Winnipeg, Manitoba, Canada
Paris Cedex 14, Paris, France
Bochum, Nordrhein Westfalen, Germany
Toulouse, Haute Garonne, France
Müllheim, Germany
Syracuse, New York, United States
Le Kremlin Bicêtre, île De France, France
Bad Reichenhall, Bayern, Germany
Düsseldorf, Nordrhein Westfalen, Germany
Oxford, Oxfordshire, United Kingdom
Dundee, Tayside Region, United Kingdom
Syracuse, New York, United States
Toulouse, Haute Garonne, France
Paris Cedex 14, Paris, France
Le Kremlin Bicêtre, île De France, France
Bad Reichenhall, Bayern, Germany
Würzburg, Bayern, Germany
Bochum, Nordrhein Westfalen, Germany
Düsseldorf, Nordrhein Westfalen, Germany
Müllheim, Germany
Bristol, Avon, United Kingdom
Toulouse Cedex 9, Haute Garonne, France
Lille Cedex, Nord, France
Poitiers Cedex, Vienne, France
Stanmore, Middlesex, United Kingdom
Sheffield, South Yorkshire, United Kingdom
Milan, Milano, Italy
Le Kremlin Bicêtre, France
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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