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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01528917
Other study ID # LAL-2-NH01
Secondary ID
Status Completed
Phase N/A
First received February 1, 2012
Last updated July 19, 2016
Start date June 2011
Est. completion date May 2013

Study information

Verified date April 2013
Source Alexion Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority United States: Institutional Review BoardUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyCanada: Health CanadaFrance: Conseil National de l'Ordre des MédecinsCzech Republic: State Institute for Drug ControlItaly: National Bioethics CommitteePoland: National Institute of Medicines
Study type Observational

Clinical Trial 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).


Description:

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.


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Study Design

Observational Model: Cohort, Time Perspective: Retrospective


Related Conditions & MeSH terms


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Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada,  Czech Republic,  France,  Italy,  Poland,  Switzerland,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Clinical History Summary Characterize patient demographic data and clinical course of disease using descriptive statistics. Expected average of 15 years No
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