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By invitation only NCT02306720

Registry of Patients With Hypophosphatasia

Conditions: Hypophosphatasia (HPP)

Sponsor: Alexion Pharmaceuticals, Inc.

trial.available_in: БГ
Overview
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.
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
Who can participate
Inclusion Criteria: * Male and female participants, of any age, with a confirmed diagnosis of HPP. * Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation. * Participant 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. 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.
Locations 10
Australia (1)
Research Site
Clayton , Victoria
Canada (1)
Research Site
Winnipeg , Manitoba
France (1)
Research Site
Toulouse , Haute Garonne
Germany (1)
Research Site
Bad Reichenhall , Bavaria
Italy (1)
Research Site
Milan , Milano
Poland (1)
Research Site
Lodz
Saudi Arabia (1)
Research Site
Riyadh
Spain (1)
Research Site
Madrid
United Kingdom (1)
Research Site
Bristol , Avon
United States (1)
Research Site
Centennial , Colorado
Technical details
Status
By invitation only
Study type
OBSERVATIONAL
Sex
Male and female
Healthy volunteers
No
Start date
20.01.2015
Completion date
31.12.2031
Registry ID
NCT02306720
Source
anzctr
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