Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
This trial is active, not recruiting.
|Sponsor||Alexion Pharma International Sarl|
|Start date||April 2010|
|End date||December 2015|
|Trial size||12 participants|
|Trial identifier||NCT01203826, ENB-008-10|
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed the ENB-006-09 study.
|United States||No locations recruiting|
|Other Countries||No locations recruiting|
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
asfotase alfa 6 mg/kg/week SC injection
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
Male or female participants from 5 years up to 13 years old.
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 involving an investigational drug, device, or treatment for HPP
|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)|
|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.|
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