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

Trial ID or NCT#

NCT01757184

Status

not recruiting iconNOT RECRUITING

Purpose

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

Eligibility Criteria

Ages Eligible for Study: Older than 4 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: Yes
Inclusion Criteria:
  1. * 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:
  1. * 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.

Investigator(s)

Gregory Enns

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Contact

Greg Enns, MD
(650) 498-5798