Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)

Registry ID

EUCTR2017-001831-38

Source registry

EUCTR

Phase

PHASE2

Sponsor

Alexion Pharma GmbH

Last update

2026-08-16

Conditions

• Hypophosphatasia

Summary

To determine the following: - Effect of asfotase alfa on reduction in plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5’-phosphate (PLP) - Tolerability of daily subcutaneous (SC) injections of asfotase alfa

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• Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
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• A Prospective Sub-Study of the Global Hypophosphatasia Registry
• Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
• Characteristics of Hypophosphatasia in Adult Patients in Rheumatology and Their Value in Developing an Algorithm to HPP-diagnosis - the COHIR Multi-center Study

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