Hypophosphatasia (HPP) Clinical Trial
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)
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
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 limited data available on the natural course of this disease
over time, particularly in patients with the juvenile-onset form.
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| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT00952484 -
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
|
Phase 2 | |
| 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)
|
||
| Enrolling by invitation |
NCT02306720 -
Registry of Patients With Hypophosphatasia
|
||
| 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 |