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

Wolman Disease Clinical Trial

Official title:
A Historical Chart Review and Longitudinal Follow-Up of Identified Patients With Wolman Disease or Cholesteryl Ester Storage Disease, Lysosomal Acid Lipase Deficiency

Clinical Trial Summary

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".

Clinical Trial Description

This is a single institution historical cohort study of patients with Wolman (WD) or Cholesteryl Ester Storage Disease (CESD). Retrospective data will be collected and abstracted from the medical records of both living and deceased patients. Additionally prospective data from living patients will be collected and abstracted annually until the end of the study. Literature sources will be used as secondary source data and will be screened to minimize/eliminate duplicative reports. ;

Study Design

Observational Model: Cohort

Related Conditions & MeSH terms

  • Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
  • Cholesterol Ester Storage Disease
  • Wolman Disease
Clinical Trial Details
NCT number NCT01884220
Study type Observational
Source Children's Hospital Medical Center, Cincinnati
Contact
Status Completed
Phase N/A
Start date November 2010
Completion date May 2014
View Details
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