NCT02112994

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

A Multi-Center, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 8 Months
• Healthy volunteers: Not accepted

Summary

This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).

Detailed description

The primary objective of this study was to evaluate the safety of intravenous (IV) infusions of sebelipase alfa in a more broad population of LAL-D participants than previously studied. Such participants may have been excluded from enrollment in other studies of LAL-D because of age, disease progression, previous treatment by hematopoietic stem cell or liver transplantation, less common disease manifestations, or disease characteristics that would preclude participation in a placebo-controlled study. This open-label study included infants \>8 months, children, and adults. At least 4 participants in the study were to be between the age of 2 and 4 years. Eligible participants received sebelipase alfa at a dose of 1 milligram/kilogram (mg/kg) every other week (qow).

Conditions

• Lysosomal Acid Lipase Deficiency

Interventions

Type	Name	Description
DRUG	Sebelipase Alfa	IV infusion of sebelipase alfa

Timeline

• Start date: 2014-06-24
• Primary completion: 2017-12-28
• Completion: 2017-12-28
• First posted: 2014-04-14
• Last updated: 2019-12-04
• Results posted: 2019-01-15

Locations

19 sites across 15 countries: United States, Australia, Belgium, Brazil, Canada, Croatia, Denmark, Germany, Italy, Mexico, Netherlands, Russia, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study