Hypophosphatasia (HPP) Clinical Trial
Official title:
A Multicenter, Open-Label Study of the Safety, Tolerability and Pharmacology of Asfotase Alfa in up to 10 Severely Affected Patients With for the Treatment of Severely Affected Patients With Infantile Hypophosphatasia (HPP)
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
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Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
|
Phase 2 | |
| Active, not recruiting |
NCT04181164 -
Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
|
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| Completed |
NCT02104219 -
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
|
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| Completed |
NCT01419028 -
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
|
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| 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
|