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Clinical Trial Summary

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


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02104219
Study type Observational
Source Alexion Pharmaceuticals
Contact
Status Completed
Phase
Start date March 2014
Completion date September 2014

See also
  Status Clinical Trial Phase
Completed NCT00952484 - Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP) Phase 2
Completed NCT00744042 - Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP) Phase 1/Phase 2
Active, not recruiting NCT04181164 - Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
Enrolling by invitation NCT02306720 - Registry of Patients With Hypophosphatasia
Completed NCT01419028 - A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
Completed NCT00739505 - Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP) Phase 1
Completed NCT01203826 - Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP) Phase 2