Status:
COMPLETED
Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Sciences (NCATS)
Conditions:
Wolman Disease
Cholesterol Ester Storage Disease
Eligibility:
All Genders
Brief 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 trea...
Detailed 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 reco...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- 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
Exclusion
Key Trial Info
Start Date :
November 1 2010
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 1 2014
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT01884220
Start Date
November 1 2010
End Date
May 1 2014
Last Update
July 28 2015
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229