Hypophosphatasia Clinical Trial
Official title:
A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging, Concurrent Control Study of the Safety, Efficacy, Pharmacokinetic of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Adolescents and Adults With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study was to test the safety and efficacy of two doses of the study drug called asfotase alfa as compared to a control group to see effects on adolescents and 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 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 |
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Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
|
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| Recruiting |
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Natural History Study of Patients With Hypophosphatasia (HPP)
|
||
| Completed |
NCT02291497 -
Burden of Disease in Hypophosphatasia (HPP)
|
N/A | |
| Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
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||
| Active, not recruiting |
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Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
|
||
| Not yet recruiting |
NCT05596539 -
Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
|
||
| Completed |
NCT02796885 -
Characterisation of Adult-Onset Hypophosphatasia
|
||
| Completed |
NCT02751801 -
Health Burden of Hypophosphatasia
|
||
| Completed |
NCT05890794 -
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
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Phase 1/Phase 2 | |
| Recruiting |
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Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
|
Phase 3 | |
| Recruiting |
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A Prospective Sub-Study of the Global Hypophosphatasia Registry
|
||
| Completed |
NCT02797821 -
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
|
Phase 2 | |
| Completed |
NCT04925804 -
Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)
|
||
| Completed |
NCT02531867 -
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
|
Phase 4 | |
| Completed |
NCT01406977 -
Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)
|
Phase 2 | |
| Recruiting |
NCT01793168 -
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
|
||
| Completed |
NCT01176266 -
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
|
Phase 2/Phase 3 | |
| Withdrawn |
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Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
|
Phase 2 | |
| Active, not recruiting |
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The PORTRAIT Study
|
||
| Recruiting |
NCT06079281 -
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
|
Phase 3 |