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
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
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

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