Hypophosphatasia Clinical Trial
Official title:
An Observational, Longitudinal Study to Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Pediatric-Onset Hypophosphatasia
Hypophosphatasia is a rare inherited metabolic disorder due to inactivating mutations of the ALPL-Gene. Particularly among adult patients, clinical manifestation exhibits a broad range of signs and symptoms, most commonly associated with musculoskeletal disabilities and compromised quality of life. Enzyme replacement therapy with Asfotase alfa (AA) is available and approved for patients with pediatric onset of the disease. This single-center observational cohort study aims at collecting clinical routine data regarding the course treatment, quality of life and physical performance in patients treated with Asfotase alfa in line with the label for pediatric-onset hypophosphatasia.
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Status | Clinical Trial | Phase | |
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Recruiting |
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Completed |
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N/A | |
Enrolling by invitation |
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Phase 2 | |
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Phase 2 | |
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Completed |
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Phase 4 | |
Completed |
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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Withdrawn |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 |