Trial Search Results

A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency/ ARISE (Acid Lipase Replacement Investigating Safety and Efficacy)

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.

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Alexion Pharmaceuticals

Stanford Investigator(s):


  • Drug: SBC-102 [sebelipase alfa] (1 mg/kg)
  • Drug: Placebo


Phase 3


Inclusion Criteria:

   - 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

   - 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

Exclusion Criteria:

   - Severe hepatic dysfunction (Child-Pugh Class C)

   - Other medical conditions or comorbidities which, in the opinion of the Investigator,
   would interfere with study compliance or data interpretation

   - Previous hematopoietic or liver transplant procedure

   - Received treatment with high-dose corticosteroids (acute or chronic) within 26 weeks.
   (Note: Subjects receiving maintenance therapy with low-dose oral, intranasal, topical,
   or inhaled corticosteroids are considered eligible for the study)

   - Known hypersensitivity to eggs

   - Participated in a study employing an investigational medicinal product within 4 weeks
   prior to randomization

Ages Eligible for Study

4 Years - N/A

Genders Eligible for Study


Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Greg Enns, MD
(650) 498-5798