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

Trial end date:
Target enrollment:
Participant gender:
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.
Phase 3
Accepts Healthy Volunteers?
Lead Sponsor:
Alexion Pharmaceuticals
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.