Hypophosphatasia (HPP) Clinical Trial
Official title:
A Multicenter, Open-Label, Dose Escalating Study of the Safety, Tolerability and Pharmacology of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)
This clinical trial studies the safety, tolerability, and pharmacology of asfotase alfa when given to adults with HPP.
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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| 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)
|
||
| Completed |
NCT01419028 -
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
|
||
| 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
|