Status:
COMPLETED
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
Lead Sponsor:
Dr. Lothar Seefried
Collaborating Sponsors:
Alexion Pharmaceuticals, Inc.
Conditions:
Hypophosphatasia
Eligibility:
All Genders
18+ years
Brief Summary
Hypophosphatasia is a rare inherited metabolic disorder due to inactivating mutations of the ALPL-Gene. Particularly among adult patients, clinical manifestation exhibits a broad range of signs and sy...
Eligibility Criteria
Inclusion
- Age ≥ 18 years at the time of enrollment
- Clinical diagnosis of pediatric-onset HPP based on low ALP (age- and sex-adjusted) and/or genetic confirmation of ALPL mutation(s), and clinical symptoms consistent with HPP
- Participant currently receiving commercial asfotase alfa treatment for HPP at the Orthopedic Institute of the Julius-Maximilians-University Würzburg, as per standard of care
- Willingness to participate in the study
- Signed informed consent
Exclusion
- Unwillingness / anticipated inability to attend further visits
- Off-label treatment with asfotase alfa
- Current participation in an Alexion Sponsored Trial
- Experimental drug/treatment
Key Trial Info
Start Date :
September 5 2018
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 31 2021
Estimated Enrollment :
23 Patients enrolled
Trial Details
Trial ID
NCT03418389
Start Date
September 5 2018
End Date
May 31 2021
Last Update
July 12 2022
Active Locations (1)
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1
Clinical Trial Unit, Orthopedic Department, Wuerzburg University
Würzburg, Germany, 97074