Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Phase 1/2 Completed 9 enrolled Alexion Pharmaceuticals, Inc.

Non-randomizedSingle-groupOpen-labelTreatment

Summary

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.

Timeline

Start: 2011-04-25
Primary completion: 2012-01-06
Completion: 2012-01-06

Publications

Results Balwani M, Breen C, Enns GM, Deegan PB, Honzik T, Jones S, Kane JP, Malinova V, Sharma R, Stock EO, Valayannopoulos V, Wraith JE, Burg J, Eckert S, Schneider E, Quinn AG. Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease. Hepatology. 2013 Sep;58(3):950-7. doi: 10.1002/hep.26289. Epub 2013 Mar 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	sebelipase alfa	Protein / enzyme biologic	0.35 mg/kg	—
Subject	sebelipase alfa	Protein / enzyme biologic	1 mg/kg	—
Subject	sebelipase alfa	Protein / enzyme biologic	3 mg/kg	—

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Summary

Timeline

Publications

Drugs

Indications