Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
Completed
Alexion Pharmaceuticals
Phase 1/Phase 2
2011-04-25
This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal
Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult
participants with liver dysfunction due to LAL Deficiency and was designed to examine 3 doses
of sebelipase alfa. The targeted number for this study was 9 evaluable participants.
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Completed
Alexion Pharmaceuticals
Phase 2/Phase 3
2011-05-04
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102
(sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL)
Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for
up to 5 years.
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
Completed
Alexion Pharmaceuticals
Phase 3
2013-01-22
This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg)
intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow)
in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester
storage disease [CESD]).
Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia.
There is currently no standard treatment for LAL-D other than supportive care. Enzyme
replacement therapy may be a potential new treatment option for LAL-D participants.
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