Trials / Completed
CompletedNCT01358370
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 40 (actual)
- Sponsor
- Alexion Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.
Detailed description
The objective of this study is to characterize key aspects of the clinical course of LAL deficiency/Wolman phenotype in patients including, but not limited to, survival and growth parameters, to serve as a historical control to inform the evaluation and care of affected patients and to provide a reference for efficacy studies of enzyme replacement or other novel therapies.
Conditions
Timeline
- Start date
- 2010-11-01
- Primary completion
- 2013-01-01
- Completion
- 2013-03-01
- First posted
- 2011-05-23
- Last updated
- 2016-06-27
Locations
14 sites across 5 countries: United States, Canada, France, Italy, United Kingdom
Source: ClinicalTrials.gov record NCT01358370. Inclusion in this directory is not an endorsement.