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

NCT01757184 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 66

Last updated 2020-12-29

Study results available
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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.

Conditions

  • Lysosomal Acid Lipase Deficiency

Interventions

DRUG

Sebelipase Alfa

IV infusions of sebelipase alfa

DRUG

Placebo

IV infusions of matched placebo

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Florian Abel, MD · Alexion Pharmaceuticals, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-01-22
Primary Completion
2014-05-30
Completion
2018-12-11

Countries

  • United States
  • Argentina
  • Australia
  • Croatia
  • Czechia
  • France
  • Germany
  • Italy
  • Japan
  • Mexico
  • Poland
  • Russia
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01757184 on ClinicalTrials.gov