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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Extension Study of Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With

Brief Summary: This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed the ENB-006-09 study.

Detailed Summary:

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.


Sponsor: Alexion Pharma GmbH

Current Primary Outcome: Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C)scale compared to baseline of treatment in ENB-006-09. [ Time Frame: 60 months ]

The time points will be pre-treatment (Baseline from the 006 study) to Month 60 of the 008 study which represents 66 months of treatment.


Original Primary Outcome: Skeletal radiograph using a qualitative Radiographic Global Inpression of Change (RGI-C)scale compared to historical controls [ Time Frame: 12 months ]

Current Secondary Outcome:

Original Secondary Outcome: PK using serum peak and trough levels and PD of Plasma PPi, PLP and serum PTH as biomarkers for HPP [ Time Frame: 12 months ]

Information By: Alexion Pharma GmbH

Dates:
Date Received: September 15, 2010
Date Started: April 2010
Date Completion:
Last Updated: August 1, 2016
Last Verified: August 2016