An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
Status: | Completed |
---|---|
Conditions: | Endocrine |
Therapuetic Areas: | Endocrinology |
Healthy: | No |
Age Range: | 5 - Any |
Updated: | 7/21/2016 |
Start Date: | June 2011 |
End Date: | May 2013 |
An Observational Study of the Clinical Characteristics and Disease Progression of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
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).
onset Lysosomal Acid Lipase (LAL) Deficiency/ Cholesteryl Ester Storage Disease (CESD).
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.
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.
Inclusion Criteria:
- Patients with late onset LAL Deficiency/ Cholesteryl Ester Storage Disease (CESD) who
are 5 years of age or older and have required data points in their medical record
Exclusion Criteria:
- Required data points for inclusion are not available
We found this trial at
7
sites
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Click here to add this to my saved trials
Stanford University Stanford University, located between San Francisco and San Jose in the heart of...
Click here to add this to my saved trials
Click here to add this to my saved trials