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
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Condition or disease |
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Hypophosphatasia (HPP) |
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 |
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 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.

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, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Probability Sample |
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. Patient must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
Exclusion Criteria:
Currently participating in an Alexion-sponsored clinical trial . Enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.

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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Alexion Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT02306720 |
Other Study ID Numbers: |
ALX-HPP-501 |
First Posted: | December 3, 2014 Key Record Dates |
Last Update Posted: | April 22, 2022 |
Last Verified: | April 2022 |
Hypophosphatasia Metal Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases |