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

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02306720
First Posted: December 3, 2014
Last Update Posted: August 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Alexion Pharmaceuticals
November 26, 2014
December 3, 2014
August 17, 2017
December 2014
December 2019   (Final data collection date for primary outcome measure)
  • 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)

Same as current
Complete list of historical versions of study NCT02306720 on ClinicalTrials.gov Archive Site
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An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.
Observational [Patient Registry]
Observational Model: Case-Control
Time Perspective: Prospective
5 Years
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Probability Sample
Patients with Hypophosphatasia (HPP)
Hypophosphatasia (HPP)
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Enrolling by invitation
500
December 2019
December 2019   (Final data collection date for primary outcome measure)

Inclusion Criteria:

Male and female patients, of any age, with a confirmed diagnosis of HPP. Patient or legal representative is able to read and/or understand the informed consent and study questionnaires in the local language.

Signed informed consent and medical records release by the patient or legal representative. Patient or patient's parent/legal representative must be willing and able to give written informed consent, and the patient must be willing to give written informed assent, if appropriate and required by local regulations.

Exclusion Criteria:

Currently participating in an Alexion-sponsored clinical trial or previously received asfotase alfa. Enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.

Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Canada,   France,   Germany,   Ireland,   Italy,   Portugal,   Russian Federation,   Saudi Arabia,   Spain,   United Kingdom,   United States
 
 
NCT02306720
ALX-HPP-501
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Alexion Pharmaceuticals
Alexion Pharmaceuticals
Not Provided
Not Provided
Alexion Pharmaceuticals
August 2017