Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||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)|
- Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09. [ Time Frame: At least 72 months of treatment with asfotase alfa ]
Evaluation of radiographic change in rickets severity (as assessed by skeletal radiographs of the hands/wrists and knees) from the Baseline of Study ENB-006-09 relative to the End of Study (EOS) visit in Study ENB-008-10 using an ordinal RGI-C scale score. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP associated rickets) to +3 (indicative of complete or near complete healing of HPP associated rickets).
The time points will be pre-treatment (Baseline from Study ENB-006-09) to the last radiographic assessment in Study ENB-008-10, which represents at least 72 months of treatment.
|Study Start Date:||April 2010|
|Study Completion Date:||June 2016|
|Primary Completion Date:||June 2016 (Final data collection date for primary outcome measure)|
Experimental: asfotase alfa
asfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection
Biological: Asfotase Alfa
Other Name: human recombinant tissue nonspecific 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|
|Shriners Hospital for Children|
|Saint Louis, Missouri, United States, 63110|
|Children's Hospital Health Sciences Centre|
|Winnipeg, Manitoba, Canada, R3A 1S1|