Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

Extension Study of Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of Asfotase Alfa (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP)

This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).

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.

Inclusion Criteria:

• Compliant and satisfactory completion of Enobia-sponsored clinical trial ENB-006-09
• Written informed consent by parent or other legal guardian prior to any study procedures being performed
• Parent or other legal guardian willing to comply with study requirements

Exclusion Criteria:

• Clinically significant disease that precludes study participation, in the Investigator's opinion
• Treatment with an investigational drug other than asfotase alfa
• Enrollment in any study (other than ENB-006-09) involving an investigational drug, device, or treatment for HPP
• Prior treatment with bisphosphonates