Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
NCT ID: NCT04195763
Last Updated: 2024-07-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
50 participants
OBSERVATIONAL
2019-11-06
2024-04-12
Brief Summary
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Detailed Description
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This is an observational study and no intervention will be administered. Participants will be treated in accordance with standard of care.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Participants with Pediatric-onset HPP
Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource.
asfotase alfa
This is an observational study and no intervention will be administered. All participants will be treated by their physician in accordance with standard of care. All medications are commercially available and will be used as directed by the treating physician.
Interventions
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asfotase alfa
This is an observational study and no intervention will be administered. All participants will be treated by their physician in accordance with standard of care. All medications are commercially available and will be used as directed by the treating physician.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Clinical diagnosis of pediatric-onset HPP
* Naïve to asfotase alfa
* Expected to begin treatment with asfotase alfa for HPP
* Registered in OneSource
* Willing and able to provide voluntary, verbal informed consent to participate in this study
Exclusion Criteria
* Unable to speak and understand English
* Unable or unwilling to complete the study surveys via telephone interview at the protocol-required time points
18 Years
99 Years
ALL
No
Sponsors
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Xcenda, LLC
UNKNOWN
Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Xcenda, LLC
Palm Harbor, Florida, United States
Countries
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Other Identifiers
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ALX-HPP-503
Identifier Type: -
Identifier Source: org_study_id
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