Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency


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Trial ID: NCT01757184


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.

Official Title

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

Stanford Investigator(s)


Inclusion Criteria:

   - Participant and/or participant's parent or legal guardian provided informed consent.

   - Participant was ≥ 4 years of age on the date of informed consent.

   - Deficiency of LAL enzyme activity confirmed by dried blood spot testing at screening.

   - Alanine aminotransferase ≥ 1.5x upper limit of normal on 2 consecutive screening
   measurements obtained at least 1 week apart.

   - Female participants of childbearing potential must not have been pregnant or
   breastfeeding and must have agreed to use a medically acceptable method of preventing
   contraception from screening until 4 weeks after the last dose of study drug.

   - Participant receiving lipid-lowering therapies must have been on a stable dose of the
   medication for at least 6 weeks prior to randomization and was willing to remain on a
   stable dose for at least the first 32 weeks of treatment in the study.

   - Participant receiving medications for the treatment of nonalcoholic fatty liver
   disease must have been on a stable dose for at least 16 weeks prior to randomization
   and was willing to remain on a stable dose for at least the first 32 weeks of
   treatment in the study.

Exclusion Criteria:

   - Severe hepatic dysfunction (Child-Pugh Class C).

   - Other medical conditions or comorbidities, which, in the opinion of the Investigator,
   would have interfered 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: Participants receiving maintenance therapy with low-dose oral, intranasal,
   topical, or inhaled corticosteroids were considered eligible for the study).

   - Known hypersensitivity to eggs.

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


drug: Placebo

drug: Sebelipase Alfa


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Contact Information

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