CompletedPhase 3

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

United States66 participantsStarted 2013-01-22

Plain-language summary

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.

Who can participate

Age range4 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 chro…

What they're measuring

Percentage Of Participants Achieving Alanine Aminotransferase Normalization

Timeframe: Double-blind Period: Baseline to the end of the Double-blind Period (Week 20). Open-label Period: Baseline to the last Open-label assessment (up to Week 256)

Trial details

NCT IDNCT01757184

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2014-05-30

Results submitted2016-01-14