Overview

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