Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
About this trial
This is an interventional treatment trial for Cholesterol Ester Storage Disease(CESD) focused on measuring Enzyme Replacement Therapy (ERT), Lysosomal Storage Disease, Late Onset Lysosomal Acid Lipase (LAL) Deficiency, Acid cholesteryl ester hydrolase deficiency, type 2, Acid lipase disease, Cholesterol ester hydrolase deficiency, LAL Deficiency, LIPA Deficiency, Wolman disease
Eligibility Criteria
Inclusion Criteria:
- Male or female participants ≥ 18 and ≤ 65 years of age
- Documented decreased LAL activity
- Evidence of liver involvement
Exclusion Criteria:
- Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstances
- Clinically significant abnormal values on laboratory screening tests, other than liver function or lipid panel tests
- Aspartate aminotransferase and/or alanine aminotransferase persistently elevated > 3x upper limit of normal at screening
- Previous hemopoietic bone marrow or liver transplant
- Current history of alcohol abuse
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
Sebelipase alfa 0.35 mg/kg
Sebelipase alfa 1 mg/kg
Sebelipase alfa 3 mg/kg
Cohort 1: Participants were administered once weekly (qw) infusions of 0.35 mg/kg sebelipase alfa.
Cohort 2: Participants were administered qw infusions of 1 mg/kg sebelipase alfa.
Cohort 3: Participants were administered qw infusions of 3 mg/kg sebelipase alfa.