Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
Status:
Completed
Trial end date:
2018-12-11
Target enrollment:
Participant gender:
Summary
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.