A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
Completed
Conditions studied: Lysosomal Acid Lipase Deficiency, Wolman Disease
In brief
This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.
Key facts
- Study ID
- NCT01358370
- Run by
- Alexion Pharmaceuticals, Inc.
- People needed
- 40
- Starts
- 2010-11-01
- Expected to finish
- 2013-03-01
- Last updated by the study team
- 2016-06-27
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Deceased patients diagnosed with LAL deficiency/Wolman phenotype in 1985 or later provided they have required data points in their medical records.
You may not qualify if…
- Patients will be excluded from the study if the required data points for inclusion are not available.
- Living LAL deficiency/Wolman phenotype patients will be excluded
Where it is running
- Cedars-Siani Medical Center — Los Angeles, California, United States
- Stanford University — Palo Alto, California, United States
- University of Minnesota — Minneapolis, Minnesota, United States
- Columbia University — New York, New York, United States
- Ney York Presbyterian Hosptial — New York, New York, United States
- North Shore Long Island Jewish Medical Center — New York, New York, United States
- Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- The Hospital for Sick Children — Toronto, Ontario, Canada
- Hospital Necker- Enfants Malades — Paris, France
- Instituto Giannina Gaslini- Ospedale Pediatrico IRCCS — Genova, Italy
- University of Turin — Turin, Italy
- Birmingham Children's Hospital NHS Foundation Trust — Birmingham, United Kingdom
- Kings College London — London, United Kingdom
- Manchester Children's Hospital — Manchester, United Kingdom
Full record on ClinicalTrials.gov
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