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

Study Identifier:
LAL-CL02
ClinicalTrials.gov Identifier:
NCT01757184
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: Sebelipase Alfa
  • Drug: Placebo
Date
Jan 2013 - May 2014
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 4+ years
Requirements Information
Inclusion and Exclusion Criteria
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.
Sex
Female & Male
Age
4+ years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: Sebelipase Alfa
  • Drug: Placebo
Date
Jan 2013 - May 2014
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 4+ years years
Requirements Information
Inclusion and Exclusion Criteria
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.

Protocol 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.

Trial Locations

Location
Status
Location
Tucson, Arizona, United States, 85724
Status
N/A
Location
Palo Alto, California, United States, 94304
Status
N/A
Location
San Francisco, California, United States, 94143-0214
Status
N/A
Location
Wilmington, Delaware, United States, 19803
Status
N/A
Location
Chicago, Illinois, United States, 60611-2605
Status
N/A
Location
Boston, Massachusetts, United States, 02115
Status
N/A
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