Registry of Patients With Hypophosphatasia

Condition(s) studied

Study summary

In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.

Eligibility

Primary outcome measure(s)

• Natural History Information — 1 Year
  To collect information on the natural history of HPP from patients of all ages, including pediatric patients and adults with HPP, regardless of age at onset.
• Burden of Disease/Patient-reported Outcomes — 1 year
  Age-appropriate PRO data will be collected using instruments to asses below: Pain, Motor Capacity, Functional Status/Disability, including Activities of Daily Living (ADL), Quality of Life (QoL)
• Characterize the epidemiology of the HPP population. — 1 year
  To characterize the epidemiology of the HPP population. Inclusion of all classifications of HPP is planned: pediatric-onset (perinatal-, infantile-, and juvenile-onset), adult-onset, benign perinatal, and odontohypophosphatasia.
• Long-Term Safety and Effectiveness of Asfotase Alfa — 1 year
  To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.

Trial sites (66)

+ 26 more sites — see the full list on the official registry below.

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