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

Completed · Phase 3 · Has a placebo group

Conditions studied: Lysosomal Acid Lipase Deficiency

In brief

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.

Key facts

Study ID

NCT01757184

Run by

Alexion Pharmaceuticals, Inc.

People needed

66

Starts

2013-01-22

Expected to finish

2018-12-11

Last updated by the study team

2020-12-29

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Study site — Tucson, Arizona, United States
• Study site — Palo Alto, California, United States
• Study site — San Francisco, California, United States
• Study site — Wilmington, Delaware, United States
• Study site — Chicago, Illinois, United States
• Study site — Boston, Massachusetts, United States
• Study site — Buffalo, New York, United States
• Study site — Manhasset, New York, United States
• Study site — New York, New York, United States
• Study site — Cincinnati, Ohio, United States
• Study site — Philadelphia, Pennsylvania, United States
• Study site — Córdoba, Argentina
• Study site — Brisbane, Australia
• Study site — New Lambton, Australia
• Study site — Parkville, Australia
• Study site — Perth, Australia
• Study site — Zagreb, Croatia
• Study site — Olomouc, Czechia
• Study site — Prague, Czechia
• Study site — Paris, France
• Study site — Vandœuvre-lès-Nancy, France
• Study site — Freiburg im Breisgau, Germany
• Study site — Mainz, Germany
• Study site — Munich, Germany
• Study site — Bergamo, Italy

Full record on ClinicalTrials.gov

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