Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed the ENB-006-09 study.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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 Hypophosphatasia (HPP)|
- 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 ] [ Designated as safety issue: No ]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.
|Study Start Date:||April 2010|
|Estimated Study Completion Date:||December 2015|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
Experimental: asfotase alfa
asfotase alfa 6 mg/kg/week SC injection
Biological: Asfotase Alfa
Other Name: human recombinant tissue non-specific alkaline phosphatase fusion protein
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01203826
|United States, Missouri|
|Shriner's Hospital for Children|
|St. Louis, Missouri, United States, 63131|
|Children's Hospital Health Sciences Centre|
|Winnipeg, Manitoba, Canada, R3B 0A6|