What is the HPP Registry?
The HPP Registry (US National Institutes of Health ClinicalTrials.gov Identifier: NCT02306720; EU PAS number: EUPAS13514) is a multinational, multicenter, observational, prospective, long-term registry designed to collect data on hypophosphatasia (HPP) epidemiology, disease history, clinical course, symptoms (including systemic aspects of disease), and burden of disease from patients of all ages who have a diagnosis of HPP. The HPP Registry was initiated in 2014 with the first patient enrolled in 2015. Data are collected in the registry from medical records as per standard of care.
The HPP Registry fulfills post-marketing regulatory requirements by providing follow-up on patients with HPP. The HPP Registry is being conducted, in part, to fulfill a Post Authorization Safety Study requirement according to EU Directive 2001/83/EC (DIR) Art 1 (15) and DIR Art 107m-q and Commission Implementing Regulation No 520/2012 Art 36-38 (EU PAS number: EUPAS13514). Successful HPP Registry implementation is contingent on contributions from both clinical sites and Alexion.
Provide access to global, longitudinal data and increased scientific knowledge to better manage patients. Alexion provides the infrastructure while ensuring data standards and quality, scientific integrity, and providing transparency.
A single, global HPP Registry can maximize both physician and patient participation to best capture information on the disease, safety, and effectiveness data in a population with a rare disease.
Inclusion criteria
The registry enrolls male and female patients of any age, with a confirmed diagnosis of HPP regardless of treatment status. Patients must have documented ALP activity below the lower limit of normal for age and sex, or a documented ALPL variant(s). The patient or parent/legal guardian will be required to provide informed consent.
Exclusion criteria
Patients cannot be currently participating in any Alexion-sponsored clinical study at the time of enrollment.
