A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency
This Phase 3 study will evaluate the efficacy and safety of 1 mg/kg IV infusions of SBC-102 (sebelipase alfa) administered every other week in patients with late onset lysosomal acid lipase (LAL) deficiency (cholesteryl ester storage disease). Late onset LAL Deficiency is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL Deficiency other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for LAL Deficiency patients.
- drug : Placebo
- drug : SBC-102 [sebelipase alfa] (1 mg/kg)
Phase: Phase 3
Ages Eligible For Study:
- Subject and/or subject's parent or legal guardian provides informed consent - Subject is ?4 years of age - Deficiency of LAL enzyme activity confirmed by dried blood spot (DBS) testing at screening - ALT ?1.5x ULN - Female subjects of childbearing potential must not be pregnant or breastfeeding - Subjects receiving lipid-lowering therapies must be on a stable dose of the medication - Subjects receiving medications for the treatment of non-alcoholic fatty liver disease must be on a stable dose