Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

Completed

Conditions studied: Wolman Disease, Cholesterol Ester Storage Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2

In brief

The purpose of this study are: to characterize and understand the natural history of disease progression in WD and CESD, and to provide historical controls for WD and CESD for developing clinical treatment trials. The hypothesis is that the variability and clinical progression in WD and CESD is large and represents a continuum of severities from a lethal infantile to near normal adults with only "fatty livers".

Key facts

Study ID

NCT01884220

Run by

Children's Hospital Medical Center, Cincinnati

People needed

4

Starts

2010-11-01

Expected to finish

2014-05-01

Last updated by the study team

2015-07-28

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• male or female of any age;
• a clinical diagnosis of WD or CESD as defined by:
• documented LAL enzyme deficiency OR
• LAL gene mutations OR
• a clinical course and tissue biopsy consistent with CESD or WD;
• written informed consent

Where it is running

• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.