Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
1571 participants
OBSERVATIONAL
2015-01-20
2031-12-31
Brief Summary
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Detailed Description
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Evaluation of safety and effectiveness data in patients with HPP who have/are receiving treatment with Asfotase alfa
Conditions
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Study Design
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CASE_CONTROL
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* 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
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Research Site
Centennial, Colorado, United States
Research Site
Hartford, Connecticut, United States
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Tampa, Florida, United States
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Decatur, Georgia, United States
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Baltimore, Maryland, United States
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Boston, Massachusetts, United States
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Boston, Massachusetts, United States
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Minneapolis, Minnesota, United States
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Kansas City, Missouri, United States
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St Louis, Missouri, United States
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Hackensack, New Jersey, United States
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Mineola, New York, United States
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Durham, North Carolina, United States
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Cincinnati, Ohio, United States
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Columbus, Ohio, United States
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Pittsburgh, Pennsylvania, United States
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Nashville, Tennessee, United States
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Salt Lake City, Utah, United States
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Charlottesville, Virginia, United States
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Madison, Wisconsin, United States
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Clayton, Victoria, Australia
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Parkville, Victoria, Australia
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Herston, , Australia
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South Brisbane, , Australia
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Linz, , Austria
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Winnipeg, Manitoba, Canada
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Oakville, Ontario, Canada
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Ottawa, Ontario, Canada
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Toronto, Ontario, Canada
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Toulouse, Haute Garonne, France
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Lille, Nord, France
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Paris, Paris, France
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Poitiers, Vienne, France
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Lyon, , France
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Paris, , France
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Le Kremlin-Bicêtre, Île-de-France Region, France
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Bad Reichenhall, Bavaria, Germany
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Würzburg, Bavaria, Germany
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Bochum, North Rhine-Westphalia, Germany
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Düsseldorf, North Rhine-Westphalia, Germany
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Dresden, Saxony, Germany
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Berlin, , Germany
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Cologne, , Germany
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Giessen, , Germany
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Homburg, , Germany
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Mainz, , Germany
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Milan, Milano, Italy
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Florence, , Italy
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Pisa, , Italy
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Rome, , Italy
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San Giovanni Rotondo, , Italy
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Lodz, , Poland
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Warsaw, , Poland
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Riyadh, , Saudi Arabia
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Madrid, , Spain
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Bristol, Avon, United Kingdom
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London, Greater London, United Kingdom
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Leicester, Leicestershire, United Kingdom
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Stanmore, Middlesex, United Kingdom
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Oxford, Oxfordshire, United Kingdom
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Sheffield, South Yorkshire, United Kingdom
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Dundee, Tayside Region, United Kingdom
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Newcastle upon Tyne, Tyne & Wear, United Kingdom
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Birmingham, West Midlands, United Kingdom
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Belfast, , United Kingdom
Countries
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References
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Dahir KM, Seefried L, Kishnani PS, Petryk A, Hogler W, Linglart A, Martos-Moreno GA, Ozono K, Fang S, Rockman-Greenberg C. Clinical profiles of treated and untreated adults with hypophosphatasia in the Global HPP Registry. Orphanet J Rare Dis. 2022 Jul 19;17(1):277. doi: 10.1186/s13023-022-02393-8.
Dahir KM, Kishnani PS, Martos-Moreno GA, Linglart A, Petryk A, Rockman-Greenberg C, Martel SE, Ozono K, Hogler W, Seefried L. Impact of muscular symptoms and/or pain on disease characteristics, disability, and quality of life in adult patients with hypophosphatasia: A cross-sectional analysis from the Global HPP Registry. Front Endocrinol (Lausanne). 2023 Mar 27;14:1138599. doi: 10.3389/fendo.2023.1138599. eCollection 2023.
Hogler W, Linglart A, Petryk A, Kishnani PS, Seefried L, Fang S, Rockman-Greenberg C, Ozono K, Dahir K, Martos-Moreno GA. Growth and disease burden in children with hypophosphatasia. Endocr Connect. 2023 Apr 25;12(5):e220240. doi: 10.1530/EC-22-0240. Print 2023 May 1.
Hogler W, Langman C, Gomes da Silva H, Fang S, Linglart A, Ozono K, Petryk A, Rockman-Greenberg C, Seefried L, Kishnani PS. Diagnostic delay is common among patients with hypophosphatasia: initial findings from a longitudinal, prospective, global registry. BMC Musculoskelet Disord. 2019 Feb 14;20(1):80. doi: 10.1186/s12891-019-2420-8.
Kishnani PS, Martos-Moreno GA, Linglart A, Petryk A, Messali A, Fang S, Rockman-Greenberg C, Ozono K, Hogler W, Seefried L, Dahir KM. Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry. Orphanet J Rare Dis. 2024 Mar 8;19(1):109. doi: 10.1186/s13023-024-03048-6.
Martos-Moreno GA, Rockman-Greenberg C, Ozono K, Petryk A, Kishnani PS, Dahir KM, Seefried L, Fang S, Hogler W, Linglart A. Clinical Profiles of Children with Hypophosphatasia prior to Treatment with Enzyme Replacement Therapy: An Observational Analysis from the Global HPP Registry. Horm Res Paediatr. 2024;97(3):233-242. doi: 10.1159/000531865. Epub 2023 Jul 13.
Seefried L, Dahir K, Petryk A, Hogler W, Linglart A, Martos-Moreno GA, Ozono K, Fang S, Rockman-Greenberg C, Kishnani PS. Burden of Illness in Adults With Hypophosphatasia: Data From the Global Hypophosphatasia Patient Registry. J Bone Miner Res. 2020 Nov;35(11):2171-2178. doi: 10.1002/jbmr.4130. Epub 2020 Aug 10.
Kishnani PS, Seefried L, Dahir KM, Martos-Moreno GA, Linglart A, Petryk A, Mowrey WR, Fang S, Ozono K, Hogler W, Rockman-Greenberg C. New insights into the landscape of ALPL gene variants in patients with hypophosphatasia from the Global HPP Registry. Am J Med Genet A. 2024 Nov;194(11):e63781. doi: 10.1002/ajmg.a.63781. Epub 2024 Jun 17.
Kishnani PS, Seefried L, Dahir KM, Martos-Moreno GA, Hogler W, Greenberg CR, Fang S, Petryk A, Mowrey WR, Linglart A, Ozono K. Disease burden by ALPL variant number in patients with non-life-threatening hypophosphatasia in the Global HPP Registry. J Med Genet. 2025 Mar 20;62(4):249-257. doi: 10.1136/jmg-2024-110383.
Tornero C, Martos-Moreno GA, Guanabens N, Florez H, Ribera A, Aguado P. Clinical characterization and impact of hypophosphatasia in Spain: An observational analysis of the Spanish cohort included in the Global HPP Registry. Med Clin (Barc). 2025 Nov;165(5):107120. doi: 10.1016/j.medcli.2025.107120. Epub 2025 Jul 25. English, Spanish.
Kishnani PS, Seefried L, Ozono K, Martos-Moreno GA, Rockman-Greenberg C, Fowler D, Burke LK, Mowrey WR, Rush ET, Ebeling PR, Hogler W, Linglart A, Fang S, Petryk A, Dahir KM. The Global Hypophosphatasia Registry: lessons learned from a decade of real-world data. Orphanet J Rare Dis. 2025 Nov 24. doi: 10.1186/s13023-025-04129-w. Online ahead of print.
Other Identifiers
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ALX-HPP-501
Identifier Type: -
Identifier Source: org_study_id