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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Tracking Information | |||
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First Submitted Date | November 26, 2014 | ||
First Posted Date | December 3, 2014 | ||
Last Update Posted Date | April 22, 2022 | ||
Actual Study Start Date | December 31, 2014 | ||
Estimated Primary Completion Date | December 31, 2024 (Final data collection date for primary outcome measure) | ||
Current Primary Outcome Measures |
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Original Primary Outcome Measures |
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Change History | |||
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 | Registry of Patients With Hypophosphatasia | ||
Official Title | An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP) | ||
Brief Summary | In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries. | ||
Detailed Description | 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 |
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Study Type | Observational [Patient Registry] | ||
Study Design | Observational Model: Case-Control Time Perspective: Prospective |
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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 * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||
Recruitment Status | Enrolling by invitation | ||
Estimated Enrollment |
900 | ||
Original Estimated Enrollment |
500 | ||
Estimated Study Completion Date | August 28, 2030 | ||
Estimated Primary Completion Date | December 31, 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. 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. |
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Sex/Gender |
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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, Italy, Poland, Russian Federation, Saudi Arabia, Spain, United Kingdom, United States | ||
Removed Location Countries | Ireland, Portugal | ||
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 | ||
Current Responsible Party | Alexion Pharmaceuticals | ||
Original Responsible Party | Same as current | ||
Current Study Sponsor | Alexion Pharmaceuticals | ||
Original Study Sponsor | Same as current | ||
Collaborators | Not Provided | ||
Investigators | Not Provided | ||
PRS Account | Alexion Pharmaceuticals | ||
Verification Date | April 2022 |