An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
NCT ID: NCT01528917
Last Updated: 2016-07-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
49 participants
OBSERVATIONAL
2011-06-30
2013-05-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
RETROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
5 Years
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Stanford University
Stanford, California, United States
Children's Memorial Hospital
Chicago, Illinois, United States
University of Minnesota
Minneapolis, Minnesota, United States
Morgan Stanley Children's Hospital of New York-Presbyterian
New York, New York, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Seattle Children's
Seattle, Washington, United States
Hospital for Sick Kids
Toronto, , Canada
1st Faculty of Medicine Charles University
Prague, , Czechia
Hôpital Necker-Enfants Malades
Paris, , France
Gaslini Institute
Genoa, , Italy
Regina Margherita Hospital
Turin, , Italy
Children's Memorial Health Institute
Warsaw, , Poland
Hopitaux Universitares De Geneve
Geneva, , Switzerland
Birmingham Children's Hospital
Birmingham, , United Kingdom
Addenbrooke's Hospital
Cambridge, , United Kingdom
Salford Royal
Salford, , United Kingdom
Countries
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References
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Burton BK, Silliman N, Marulkar S. Progression of liver disease in children and adults with lysosomal acid lipase deficiency. Curr Med Res Opin. 2017 Jul;33(7):1211-1214. doi: 10.1080/03007995.2017.1309371. Epub 2017 Apr 3.
Burton BK, Deegan PB, Enns GM, Guardamagna O, Horslen S, Hovingh GK, Lobritto SJ, Malinova V, McLin VA, Raiman J, Di Rocco M, Santra S, Sharma R, Sykut-Cegielska J, Whitley CB, Eckert S, Valayannopoulos V, Quinn AG. Clinical Features of Lysosomal Acid Lipase Deficiency. J Pediatr Gastroenterol Nutr. 2015 Dec;61(6):619-25. doi: 10.1097/MPG.0000000000000935.
Related Links
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Alexion Pharmaceuticals
Other Identifiers
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LAL-2-NH01
Identifier Type: -
Identifier Source: org_study_id
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