HPP Registry TM

The HPP Registry (US National Institutes of Health ClinicalTrials.gov Identifier: NCT02306720) 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.

The HPP Registry fulfills post-marketing regulatory requirements by providing follow-up on patients with HPP. The HPP Registry will be conducted, in part, as a Post Authorization Safety Study 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. Successful HPP Registry implementation is contingent on contributions from both academia and Alexion.

Academia provides access to global, longitudinal data and increased scientific knowledge to better manage patients. Alexion provides the infrastructure and fosters relationships with academic partners, building credibility and scientific integrity, while also providing transparency and clear publication guidelines.

A single, global HPP Registry can maximize both physician and patient participation to best capture information on the disease, safety, and efficacy data in a population with a very rare disease.

 

Inclusion criteria



The study population is male and female patients of any age, with a confirmed diagnosis of HPP (patients must have documented ALP activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation). Any patient may be eligible. 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.