Registry of Patients With Hypophosphatasia
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|ClinicalTrials.gov Identifier: NCT02306720|
Recruitment Status : Enrolling by invitation
First Posted : December 3, 2014
Last Update Posted : April 22, 2022
|Condition or disease|
The HPP Registry is an observational, prospective, long-term registry designed to collect data on HPP epidemiology, disease history, clinical course, symptoms and burden of disease from patients of all ages who have a diagnosis of HPP.
Evaluation of safety and effectiveness data in patients with HPP who have/are receiving treatment with Asfotase alfa
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||900 participants|
|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 31, 2014|
|Estimated Primary Completion Date :||December 31, 2024|
|Estimated Study Completion Date :||August 28, 2030|
- Natural History Information [ Time Frame: 1 Year ]To collect information on the natural history of HPP from patients of all ages, including pediatric patients and adults with HPP, regardless of age at onset.
- Burden of Disease/Patient-reported Outcomes [ Time Frame: 1 year ]Age-appropriate PRO data will be collected using instruments to asses below: Pain, Motor Capacity, Functional Status/Disability, including Activities of Daily Living (ADL), Quality of Life (QoL)
- Characterize the epidemiology of the HPP population. [ Time Frame: 1 year ]To characterize the epidemiology of the HPP population. Inclusion of all classifications of HPP is planned: pediatric-onset (perinatal-, infantile-, and juvenile-onset), adult-onset, benign perinatal, and odontohypophosphatasia.
- Long-Term Safety and Effectiveness of Asfotase Alfa [ Time Frame: 1 year ]To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.
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