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Clinical Trial Summary

This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.


Clinical Trial 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.

Efficacy analyses were prospectively defined in the protocol with a comparison to historical controls. The historical control group came from patients whose characteristics matched as closely as possible the entry criteria for the trial. The control group included all patients who had x-rays within the age range defined by the inclusion criteria of this study (5 to 12 years of age, inclusive, with open growth plates).

The pre-specified plan for analysis was to combine the two asfotase alfa treated groups (asfotase alfa 2 mg/kg subcutaneous (SC) injection three times per week or 3 mg/kg subcutaneous (SC) injection three times per week) and compare them to historical controls. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT00952484
Study type Interventional
Source Alexion Pharmaceuticals
Contact
Status Completed
Phase Phase 2
Start date September 2009
Completion date July 2010

See also
  Status Clinical Trial Phase
Completed NCT00744042 - Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP) Phase 1/Phase 2
Active, not recruiting NCT04181164 - Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
Completed NCT02104219 - Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
Completed NCT01419028 - A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
Completed NCT00739505 - Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP) Phase 1
Completed NCT01203826 - Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP) Phase 2
Enrolling by invitation NCT02306720 - Registry of Patients With Hypophosphatasia