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

• Status: Completed
• Trial end date: 2012-01-06
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

Criteria

Inclusion Criteria:

• Male or female participants ≥ 18 and ≤ 65 years of age

• Documented decreased LAL activity

• Evidence of liver involvement

Exclusion Criteria:

• Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstances

• Clinically significant abnormal values on laboratory screening tests, other than liver function or lipid panel tests

• Aspartate aminotransferase and/or alanine aminotransferase persistently elevated > 3x upper limit of normal at screening

• Previous hemopoietic bone marrow or liver transplant

• Current history of alcohol abuse