An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

Completed

Conditions studied: Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency

In brief

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).

Key facts

Study ID

NCT01528917

Run by

Alexion Pharmaceuticals, Inc.

People needed

49

Starts

2011-06-01

Expected to finish

2013-05-01

Last updated by the study team

2016-07-20

Who can join

Age: 5 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Required data points for inclusion are not available

Where it is running

• Stanford University — Stanford, California, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Morgan Stanley Children's Hospital of New York-Presbyterian — New York, New York, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Seattle Children's — Seattle, Washington, United States
• Hospital for Sick Kids — Toronto, Canada
• 1st Faculty of Medicine Charles University — Prague, Czechia
• Hôpital Necker-Enfants Malades — Paris, France
• Gaslini Institute — Genoa, Italy
• Regina Margherita Hospital — Turin, Italy
• Children's Memorial Health Institute — Warsaw, Poland
• Hopitaux Universitares De Geneve — Geneva, Switzerland
• Birmingham Children's Hospital — Birmingham, United Kingdom
• Addenbrooke's Hospital — Cambridge, United Kingdom
• Salford Royal — Salford, United Kingdom

Full record on ClinicalTrials.gov

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