NCT02104219

Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

A Retrospective, Non-interventional, Epidemiologic Study of the Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 32 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.

Detailed description

Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.

Conditions

• Hypophosphatasia (HPP)

Timeline

• Start date: 2014-03-01
• Primary completion: 2014-09-01
• Completion: 2014-09-01
• First posted: 2014-04-04
• Last updated: 2019-04-01
• Results posted: 2015-12-18

Locations

19 sites across 8 countries: United States, Australia, Canada, France, Netherlands, Russia, Turkey (Türkiye), United Kingdom