An observational disease and clinical outcomes registry of patients with lysosomal acid lipase (LAL) deficiency (ALX-LALD-501)

Study identification

• EU PAS number:
  EUPAS13276
• Study ID:
  47904
• Official title and acronym:
  An observational disease and clinical outcomes registry of patients with lysosomal acid lipase (LAL) deficiency (ALX-LALD-501)
• DARWIN EU® study:
  No
• Study countries:
  Australia

  Belgium

  Brazil

  Canada

  Croatia

  Czechia

  Denmark

  France

  Germany

  Greece

  Ireland

  Israel

  Italy

  Japan

  Mexico

  Netherlands

  Poland

  Portugal

  Russian Federation

  Saudi Arabia

  Slovenia

  Spain

  United Kingdom

  United States
• Study description:
  This is an observational, multi-center, international registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL deficiency.
• Study status:
  Ongoing

Study timelines

Date when funding contract was signed

Planned:30/05/2013

Actual:30/05/2013

Study start date

Planned:30/05/2013

Actual:30/05/2013

Date of final study report

Planned:30/01/2027

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Alexion Pharmaceuticals Inc.

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)