Hypophosphatasia Clinical Trial
Official title:
An Open-label, Intra-patient Dose-escalation Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP).
The purpose of the study is to determine tolerability, PK/PD and preliminary efficacy of BPS804 in adult patients with HPP treated with multiple escalating doses of BPS804. This study will allow a comparison of several doses of the study drug within the first two weeks after administration and after a longer assessment period for the highest dose level to enable selection of dose ranges to be tested in subsequent studies in the HPP indication.
This study was conducted and previously posted by Novartis. The record was transferred to Ultragenyx in February 2021. ;
| 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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| Completed |
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| Completed |
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Health Burden of Hypophosphatasia
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Characterisation of Adult-Onset Hypophosphatasia
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| Completed |
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Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
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Phase 1/Phase 2 | |
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A Prospective Sub-Study of the Global Hypophosphatasia Registry
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Phase 3 | |
| Completed |
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Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
|
Phase 2 | |
| Completed |
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Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
|
Phase 2 | |
| Completed |
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|
||
| Completed |
NCT02531867 -
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
|
Phase 4 | |
| Recruiting |
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Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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| Completed |
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|
Phase 2/Phase 3 | |
| Withdrawn |
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Phase 2 | |
| Active, not recruiting |
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| Recruiting |
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|
Phase 3 |