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

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.
ClinicalTrials.gov Identifier: NCT02306720
Recruitment Status : Enrolling by invitation
First Posted : December 3, 2014
Last Update Posted : August 17, 2017
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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

Condition or disease
Hypophosphatasia (HPP)

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

Study Type : Observational [Patient Registry]
Estimated Enrollment : 500 participants
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)
Actual Study Start Date : December 2014
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources




Primary Outcome Measures :
  1. Patient-reported Outcomes [ Time Frame: 1 year ]

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

    Pain


  2. Patient-reported Outcomes [ Time Frame: 1 year ]

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

    Motor Capacity


  3. 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)


  4. Patient-reported Outcomes [ Time Frame: 1 year ]

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

    Quality of Life (QoL)




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.


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


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Sponsors and Collaborators
Alexion Pharmaceuticals

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02306720     History of Changes
Other Study ID Numbers: ALX-HPP-501
First Posted: December 3, 2014    Key Record Dates
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