Clinical Trial: An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Observational [Patient Registry]

Official Title: An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

Brief Summary: To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.

Detailed Summary: To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome:

• Patient-reported Outcomes [ Time Frame: 1 year ]

  Age-appropriate PRO data will be collected using instruments to asses below:

  Pain

• Patient-reported Outcomes [ Time Frame: 1 year ]

  Age-appropriate PRO data will be collected using instruments to asses below:

  Motor Capacity

• Patient-reported Outcomes [ Time Frame: 1 year ]

  Age-appropriate PRO data will be collected using instruments to asses below:

  Functional Status/Disability, including Activities of Daily Living (ADL)

• Patient-reported Outcomes [ Time Frame: 1 year ]

  Age-appropriate PRO data will be collected using instruments to asses below:

  Quality of Life (QoL)

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Alexion Pharmaceuticals

Dates:
Date Received: November 26, 2014
Date Started: December 2014
Date Completion: December 2019
Last Updated: February 22, 2017
Last Verified: February 2017