NCT01205152

Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

Extension Study of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children With Hypophosphatasia (HPP)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 24 Weeks – 42 Months
• Healthy volunteers: Not accepted

Summary

This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042). Partial funding for this study was provided by the Office of Orphan Product Development (OOPD).

Detailed description

Asfotase Alfa was formerly referred to as ENB-0040 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 limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.

Conditions

• Hypophosphatasia

Interventions

Type	Name	Description
BIOLOGICAL	asfotase alfa

Timeline

• Start date: 2009-04-01
• Primary completion: 2016-08-01
• Completion: 2016-08-01
• First posted: 2010-09-20
• Last updated: 2019-03-13
• Results posted: 2017-11-17

Locations

8 sites across 3 countries: United States, United Arab Emirates, United Kingdom

Regulatory

• FDA-regulated drug study