Trials / Completed
CompletedNCT01419028
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
A Retrospective, Non-interventional Epidemiologic Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 48 (actual)
- Sponsor
- Alexion Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This study aims to characterize the natural history of patients with severe perinatal or infantile 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
Timeline
- Start date
- 2012-08-01
- Primary completion
- 2013-06-01
- Completion
- 2014-02-01
- First posted
- 2011-08-17
- Last updated
- 2019-04-01
- Results posted
- 2014-07-21
Locations
12 sites across 7 countries: United States, Australia, Canada, Germany, Spain, Taiwan, United Kingdom
Source: ClinicalTrials.gov record NCT01419028. Inclusion in this directory is not an endorsement.