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

Tracking Information
First Submitted Date	November 26, 2014
First Posted Date	December 3, 2014
Last Update Posted Date	August 17, 2017
Actual Study Start Date	December 2014
Estimated Primary Completion Date	December 2019 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures; (submitted: December 1, 2014)	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 Measures	Same as current
Change History	Complete list of historical versions of study NCT02306720 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures	Not Provided
Original Secondary Outcome Measures	Not Provided
Current Other Outcome Measures	Not Provided
Original Other Outcome Measures	Not Provided
Descriptive Information
Brief Title	An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)
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 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]
Study Design	Observational Model: Case-Control; Time Perspective: Prospective
Target Follow-Up Duration	5 Years
Biospecimen	Not Provided
Sampling Method	Probability Sample
Study Population	Patients with Hypophosphatasia (HPP)
Condition	Hypophosphatasia (HPP)
Intervention	Not Provided
Study Groups/Cohorts	Not Provided
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status	Enrolling by invitation
Estimated Enrollment; (submitted: December 1, 2014)	500
Original Estimated Enrollment	Same as current
Estimated Study Completion Date	December 2019
Estimated Primary Completion Date	December 2019 (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender	Sexes Eligible for Study: All
Ages	Child, Adult, Older Adult
Accepts Healthy Volunteers	No
Contacts	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries	Australia, Canada, France, Germany, Ireland, Italy, Portugal, Russian Federation, Saudi Arabia, Spain, United Kingdom, United States
Removed Location Countries
Administrative Information
NCT Number	NCT02306720
Other Study ID Numbers	ALX-HPP-501
Has Data Monitoring Committee	Not Provided
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Alexion Pharmaceuticals
Study Sponsor	Alexion Pharmaceuticals
Collaborators	Not Provided
Investigators	Not Provided
PRS Account	Alexion Pharmaceuticals
Verification Date	August 2017