NCT00952484

Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)

A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Historical Control Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 5 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.

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 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. Efficacy analyses were prospectively defined in the protocol with a comparison to historical controls. The historical control group came from patients whose characteristics matched as closely as possible the entry criteria for the trial. The control group included all patients who had x-rays within the age range defined by the inclusion criteria of this study (5 to 12 years of age, inclusive, with open growth plates). The pre-specified plan for analysis was to combine the two asfotase alfa treated groups (asfotase alfa 2 mg/kg subcutaneous (SC) injection three times per week or 3 mg/kg subcutaneous (SC) injection three times per week) and compare them to historical controls.

Conditions

• Hypophosphatasia (HPP)

Interventions

Type	Name	Description
BIOLOGICAL	asfotase alfa	2 mg/kg subcutaneous injection three times per week for 6 months.
BIOLOGICAL	asfotase alfa	3 mg/kg subcutaneous injection three times per week for 6 months.

Timeline

• Start date: 2009-09-01
• Primary completion: 2010-07-01
• Completion: 2010-07-01
• First posted: 2009-08-06
• Last updated: 2019-04-01
• Results posted: 2011-07-26

Locations

2 sites across 2 countries: United States, Canada