ALX-HPP-502, non-interventional study in patients with Juvenile HPP

• Research type

  Research Study

• Full title

  A RETROSPECTIVE, NON-INTERVENTIONAL, EPIDEMIOLOGIC STUDY OF THE NATURAL HISTORY OF PATIENTS WITH JUVENILE-ONSET HYPOPHOSPHATASIA (HPP)

• IRAS ID

  147095

• Contact name

  Nicholas Bishop

• Contact email

  n.j.bishop@sheffield.ac.uk

• Sponsor organisation

  Alexion Pharmaceutical Inc

• Research summary

  This study is being conducted to learn about patients who have juvenile onset Hypophosphatasia (HPP), for example their growth, and manifestations or complications of the disease. HPP is a bone disorder caused by gene mutations or changes. These gene mutations result in low levels or absence of an enzyme needed to harden children’s bones.
  This is a retrospective, non-interventional study, with no study drug or medical procedures involved.
  Study staff will review the patient’s medical records for the following, if available:
  • demographic information;
  • medical history;
  • diagnostic history including x-rays and laboratory history;
  • developmental history including growth data;

  Study staff will not have to ask the patient / patient’s parent(s) any questions regarding medical history. All data will be obtained from medical records.
  Patients / Patient’s Parent(s) will only need to attend the study site once, to provide informed consent.

• REC name

  London - Camberwell St Giles Research Ethics Committee

• REC reference

  14/LO/0248

• Date of REC Opinion

  5 Feb 2014

• REC opinion

  Favourable Opinion