NCT00744042

Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

A Multicenter, Open-Label Study of the Safety, Tolerability and Pharmacology of Asfotase Alfa in up to 10 Severely Affected Patients With for the Treatment of Severely Affected Patients With Infantile Hypophosphatasia (HPP)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 36 Months
• Healthy volunteers: Not accepted

Summary

This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with 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

• Hypophosphatasia (HPP)

Interventions

Type	Name	Description
BIOLOGICAL	asfotase alfa

Timeline

• Start date: 2008-09-01
• Primary completion: 2010-05-01
• Completion: 2010-05-01
• First posted: 2008-08-29
• Last updated: 2019-04-01
• Results posted: 2011-09-30

Locations

10 sites across 4 countries: United States, Canada, United Arab Emirates, United Kingdom