Ignite Creation Date:
2025-12-25 @ 1:43 AM
Ignite Modification Date:
2026-03-03 @ 12:38 AM
Study NCT ID:
NCT02112994
Status:
COMPLETED
Last Update Posted:
2019-12-04
First Post:
2014-03-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
Sponsor:
Alexion Pharmaceuticals, Inc.
Organization:
Conditions & Keywords Data
Conditions:
Conditions
| Condition Brief | Condition Text | View |
|---|---|---|
| None | Lysosomal Acid Lipase Deficiency | View |
Keywords:
Keywords
| Keyword Brief | Keyword Text | View |
|---|---|---|
| None | Enzyme Replacement Therapy (ERT) | View |
| None | Lysosomal Storage Disease | View |
| None | Late Onset Lysosomal Acid Lipase (LAL) Deficiency | View |
| None | Acid cholesteryl ester hydrolase deficiency, type 2 | View |
| None | Acid lipase disease | View |
| None | Cholesterol ester hydrolase deficiency | View |
| None | LAL Deficiency | View |
| None | LIPA Deficiency | View |
| None | Wolman disease | View |
| None | Additional relevant MeSH terms: | View |
| None | Cholesterol Ester Storage Disease | View |
| None | Metabolic Diseases | View |
| None | Lipidoses | View |
| None | Lipid Metabolism, Inborn Errors | View |
| None | Metabolism, Inborn Errors | View |
| None | Genetic Diseases, Inborn | View |
| None | Lysosomal Storage Diseases | View |
| None | Lipid Metabolism Disorders | View |
| None | Infant, Newborn, Diseases | View |