ALX-HPP-501 V1.1
Research type
Research Study
Full title
An Observational, Longitudinal, Prospective, Long-Term Registry of Patients with Hypophosphatasia
IRAS ID
167543
Contact name
Vrinda Saraff
Contact email
Sponsor organisation
Alexion Pharmaceuticals Ltd
Duration of Study in the UK
5 years, 0 months, 2 days
Research summary
We are conducting this study to learn about patients who have Hypophosphatasia (HPP). HPP is an ultra-rare bone disorder caused by gene mutations or changes. These gene mutations result in low or absent levels of an enzyme needed to harden bones. There is no approved treatment for this condition.
Due to the rare nature of this disease, and considering the lack of information regarding diagnosis and health care management in a “real world” setting, this study will document the demography, clinical course, and impact of disease on patients of all ages who have HPP.
There is no study drug administered in this study, patients who consent to participate will be asked to complete questionnaires at various timepoints, in line with routine clinic visits. We will be collecting data on the routine clinical care of patients of all ages, regardless of the age of onset of HPP, by reviewing patient medical records. There are no protocol specific clinical procedures to be undertaken. We plan to enroll at least 500 patients to better understand the disease. The expected duration of the study is at least 5 years.REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
14/WM/1233
Date of REC Opinion
16 Dec 2014
REC opinion
Further Information Favourable Opinion