Trials / Completed
CompletedNCT01884220
Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up
A Historical Chart Review and Longitudinal Follow-Up of Identified Patients With Wolman Disease or Cholesteryl Ester Storage Disease, Lysosomal Acid Lipase Deficiency
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 4 (actual)
- Sponsor
- Children's Hospital Medical Center, Cincinnati · Academic / Other
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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".
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 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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | There are no interventions in this study. |
Timeline
- Start date
- 2010-11-01
- Primary completion
- 2014-05-01
- Completion
- 2014-05-01
- First posted
- 2013-06-21
- Last updated
- 2015-07-28
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT01884220. Inclusion in this directory is not an endorsement.