AN OBSERVATIONAL, LONGITUDINAL, PROSPECTIVE, LONG-TERM REGISTRY OF PATIENTS WITH HYPOPHOSPHATASIA (ALX-HPP-501)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/47907
• EU PAS number:
  EUPAS13514
• Study ID:
  47907
• Official title and acronym:
  AN OBSERVATIONAL, LONGITUDINAL, PROSPECTIVE, LONG-TERM REGISTRY OF PATIENTS WITH HYPOPHOSPHATASIA (ALX-HPP-501)
• DARWIN EU® study:
  No
• Study countries:
  Australia

  Austria

  Canada

  France

  Germany

  Italy

  Japan

  Poland

  Saudi Arabia

  Spain

  United Kingdom

  United States
• Study description:
  HPP is a rare disease that has historically been largely treated symptomatically. Only one therapy designed to treat the underlying cause of the disease (Strensiq® asfotase alfa) has been approved for commercial use. Due to the rare nature of this disease, and considering the lack of information regarding diagnosis patterns and health care management in a “real world” setting, this study will collect data on epidemiology, HPP history, genetics (ALPL variants) clinical course, symptoms (including systemic aspects of the disease), and burden of disease from patients of any age who have a diagnosis of HPP, including patients who are either untreated or receiving treatment for HPP. For patients treated with asfotase alfa, the Registry collects data on asfotase alfa dosing, effectiveness of treatment, serious adverse events (SAEs), immunogenicity, pregnancy and neonatal outcome data, and pre-defined targeted events. Accordingly, the Registry will permit better delineation between the natural disease course of HPP and the disease course in patients who are treated.
• Study status:
  Ongoing

Study timelines

Date when funding contract was signed

Planned: 31/07/2014

Actual: 31/07/2014

Study start date

Planned: 20/01/2015

Actual: 20/01/2015

Date of final study report

Planned: 28/08/2030

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Alexion Pharmaceuticals, Inc.

Study protocol

Initial protocol

Asfotase alfa_ALX-HPP-501 Protocol Amendment 4.2_06May2016_Final

Updated protocol

ALX-HPP-501 PA 6.8_EU exc Germany Final_24Nov2020_signed

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)