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LAL-Deficiency clinical trials

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NCT ID: NCT01488097 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

Start date: December 12, 2011
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT01307098 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

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

Start date: April 25, 2011
Phase: Phase 1/Phase 2
Study type: Interventional

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.