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Sebelipase Alfa

DRUG5 trials

Sponsors

Alexion Pharmaceuticals, Inc.

Conditions

Cholesterol Ester Storage Disease (CESD)LAL-DeficiencyLysosomal Acid Lipase Deficiency

Phase 2

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
CompletedNCT01488097
Alexion Pharmaceuticals, Inc.Cholesterol Ester Storage Disease (CESD), LAL-Deficiency, Lysosomal Acid Lipase Deficiency
Start: 2011-12-12End: 2017-06-21Updated: 2018-07-20
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
CompletedNCT02112994
Alexion Pharmaceuticals, Inc.Lysosomal Acid Lipase Deficiency
Start: 2014-06-24End: 2017-12-28Updated: 2019-12-04
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
TerminatedNCT02193867
Alexion Pharmaceuticals, Inc.Lysosomal Acid Lipase Deficiency
Start: 2014-06-06End: 2018-10-30Updated: 2019-11-18

Phase 3

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
CompletedNCT01757184
Alexion Pharmaceuticals, Inc.Lysosomal Acid Lipase Deficiency
Start: 2013-01-22End: 2018-12-11Updated: 2020-12-29

Unknown Phase

An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
NCT02376751
Alexion Pharmaceuticals, Inc.Lysosomal Acid Lipase Deficiency
Updated: 2016-06-08

Related Papers

Long‐Term Sebelipase Alfa Treatment in Children and Adults With Lysosomal Acid Lipase Deficiency
Journal of Pediatric Gastroenterology and Nutrition2022-04-1920 citations
Sebelipase alfa in children and adults with lysosomal acid lipase deficiency: Final results of the ARISE study
Journal of Hepatology2021-11-1029 citations
Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies.
2021-01-0622 citations
Sebelipase alfa for lysosomal acid lipase deficiency: 5‐year treatment experience from a phase 2 open‐label extension study
Liver International2020-07-1322 citations
Sebelipase alfa improves atherogenic biomarkers in adults and children with lysosomal acid lipase deficiency.
2018-03-0910 citations
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
New England Journal of Medicine2015-09-10238 citations
Sebelipase Alfa Normalizes ALT and Multiple other Disease-Related Abnormalities in Patients with LAL-D
MD Conference Express2014-12-01
Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency.
2014-06-3058 citations
New Diagnostic Method for Lysosomal Acid Lipase Deficiency and the Need to Recognize Its Manifestation in Infants (Wolman Disease)
Journal of Pediatric Gastroenterology and Nutrition2013-09-1714 citations