CLINICAL TRIALS PROFILE FOR SEBELIPASE ALFA

All Clinical Trials for sebelipase alfa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01307098 ↗	Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency	Completed	Alexion Pharmaceuticals	Phase 1/Phase 2	2011-04-25	This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult participants with liver dysfunction due to LAL Deficiency and was designed to examine 3 doses of sebelipase alfa. The targeted number for this study was 9 evaluable participants.
NCT01371825 ↗	Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency	Completed	Alexion Pharmaceuticals	Phase 2/Phase 3	2011-05-04	This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.
NCT01473875 ↗	Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency	Completed	Alexion Pharmaceuticals	Phase 2/Phase 3	2011-05-04	This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.
NCT01488097 ↗	Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency	Completed	Alexion Pharmaceuticals	Phase 2	2011-12-12	This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysfunction due to lysosomal acid lipase (LAL) deficiency.
NCT01757184 ↗	Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency	Completed	Alexion Pharmaceuticals	Phase 3	2013-01-22	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.
NCT02112994 ↗	Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency	Completed	Alexion Pharmaceuticals	Phase 2	2014-06-24	This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).
NCT02193867 ↗	Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency	Terminated	Alexion Pharmaceuticals	Phase 2	2014-06-06	This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.

Clinical Trial Conditions for sebelipase alfa

Condition Name

Condition Name for sebelipase alfa
Intervention	Trials
Lysosomal Acid Lipase Deficiency	8
Wolman Disease	2
Cholesterol Ester Storage Disease (CESD)	2
Cholesterol Ester Storage Disease(CESD)	2

Condition MeSH

Condition MeSH for sebelipase alfa
Intervention	Trials
Wolman Disease	8
Cholesterol Ester Storage Disease	3
Failure to Thrive	2

Clinical Trial Locations for sebelipase alfa

Trials by Country

Trials by Country for sebelipase alfa
Location	Trials
United States	25
United Kingdom	8
France	5
Canada	4
Australia	3

Trials by US State

Trials by US State for sebelipase alfa
Location	Trials
California	6
New York	4
Pennsylvania	3
Ohio	2
Illinois	2

Clinical Trial Progress for sebelipase alfa

Clinical Trial Phase

Clinical Trial Phase for sebelipase alfa
Clinical Trial Phase	Trials
Phase 3	1
Phase 2/Phase 3	2
Phase 2	3

Clinical Trial Status

Clinical Trial Status for sebelipase alfa
Clinical Trial Phase	Trials
Completed	6
No longer available	1
Terminated	1

Clinical Trial Sponsors for sebelipase alfa

Sponsor Name

Sponsor Name for sebelipase alfa
Sponsor	Trials
Alexion Pharmaceuticals	8

Sponsor Type

Sponsor Type for sebelipase alfa
Sponsor	Trials
Industry	8