Trial ID or NCT#





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.

Official Title

A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency

Eligibility Criteria

Ages Eligible for Study: Older than 4 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No


Gregory Enns
Professor of Pediatrics (Genetics) at the Lucile Salter Packard Children's Hospital

Contact us to find out if this trial is right for you.


Greg Enns, MD
(650) 498-5798