Hypophosphatasia (HPP) Clinical Trial
Official title:
A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Historical Control Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP)
This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.
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.
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| 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
|