Status:
COMPLETED
Burden of Disease in Hypophosphatasia (HPP)
Lead Sponsor:
Wuerzburg University Hospital
Conditions:
Hypophosphatasia
Eligibility:
All Genders
18+ years
Brief Summary
Hypophosphatasia (HPP) is a rare, inherited metabolic disease caused by inactivating mutations in the Alkaline Phosphatase (ALPL) gene, coding for the Tissue-nonspecific alkaline phosphatase (TNAP). P...
Eligibility Criteria
Inclusion
- Adult males and females (age ≥ 18 years)
- Established diagnosis of Hypophosphatasia
- Reduced Serum/Plasma ALP (Alkaline phosphatase)-Activity below age and sex specific reference range of the respective test kit applied (measured at least twice with a minimum 4 week interval)
- At least one of the items below:
- Genetically secured ALPL-Mutation
- Elevated PLP (Pyridoxal 5-Phosphate) (urine or serum), above ULN (Upper level of normal)
- Symptoms of the disease
- Signed informed consent
Exclusion
- Current / previous treatment with Asfotase alfa
- Current participation in another clinical study
Key Trial Info
Start Date :
October 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 1 2016
Estimated Enrollment :
114 Patients enrolled
Trial Details
Trial ID
NCT02291497
Start Date
October 1 2014
End Date
June 1 2016
Last Update
November 8 2016
Active Locations (1)
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1
Clinical Trial Unit, Orthopaedische Klinik Koenig-Ludwig-Haus, Lehrstuhl der Universitaet Wuerzburg
Würzburg, Bavaria, Germany, 97074