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

Condition
Hypophosphatasia (HPP)

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Case-Control
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • 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)



Estimated Enrollment: 500
Actual Study Start Date: December 2014
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
Detailed Description:
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients with Hypophosphatasia (HPP)
Criteria

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.

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02306720


  Show 55 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02306720     History of Changes
Other Study ID Numbers: ALX-HPP-501
First Submitted: November 26, 2014
First Posted: December 3, 2014
Last Update Posted: August 17, 2017
Last Verified: August 2017

Additional relevant MeSH terms:
Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases