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

• ClinicalTrials.gov Identifier: NCT02306720
• Recruitment Status: Enrolling by invitation
• First Posted: December 3, 2014
• Last Update Posted: January 12, 2021
• Sponsor: Alexion Pharmaceuticals
• Information provided by (Responsible Party): Alexion Pharmaceuticals

Tracking Information

• First Submitted Date: November 26, 2014
• First Posted Date: December 3, 2014
• Last Update Posted Date: January 12, 2021
• Actual Study Start Date: December 2014
• Estimated Primary Completion Date: December 2024 (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
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified 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 *

• Seefried L, Dahir K, Petryk A, Högler W, Linglart A, Martos-Moreno GÁ, Ozono K, Fang S, Rockman-Greenberg C, Kishnani PS. Burden of Illness in Adults With Hypophosphatasia: Data From the Global Hypophosphatasia Patient Registry. J Bone Miner Res. 2020 Nov;35(11):2171-2178. doi: 10.1002/jbmr.4130. Epub 2020 Aug 10.
• Högler W, Langman C, Gomes da Silva H, Fang S, Linglart A, Ozono K, Petryk A, Rockman-Greenberg C, Seefried L, Kishnani PS. Diagnostic delay is common among patients with hypophosphatasia: initial findings from a longitudinal, prospective, global registry. BMC Musculoskelet Disord. 2019 Feb 14;20(1):80. doi: 10.1186/s12891-019-2420-8.

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 2024
• Estimated Primary Completion Date: December 2024 (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

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: January 2021