Clinical Trial: An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
Study Status: Completed
Recruit Status: Completed
Study Type: Observational
Official Title: An Observational Study of the Clinical Characteristics and Disease Progression of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotyp
Brief Summary: This is a Natural History study to characterize key aspects of the clinical course of late onset Lysosomal Acid Lipase (LAL) Deficiency/ Cholesteryl Ester Storage Disease (CESD).
Detailed Summary: The objective of this study is to characterize key aspects of the clinical presentation, disease phenotype and progression of patients with late onset Lysosomal Acid Lipase (LAL) Deficiency/ Cholesteryl Ester Storage Disease (CESD) including, but not limited to, age of presentation, onset of hepatomegaly, progression over time of liver function, and stability of lipid abnormalities.
Sponsor: Alexion Pharmaceuticals
Current Primary Outcome: Clinical History Summary [ Time Frame: Expected average of 15 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Alexion Pharmaceuticals
Dates:
Date Received: February 1, 2012
Date Started: June 2011
Date Completion:
Last Updated: July 19, 2016
Last Verified: April 2013