Overview

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

Status:
Completed
Trial end date:
2017-06-21
Target enrollment:
0
Participant gender:
All
Summary
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.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Alexion Pharmaceuticals
Criteria
Inclusion Criteria:

- Participant received all 4 scheduled doses of sebelipase alfa in Study LAL-CL01 with
no life-threatening or unmanageable study drug toxicity.

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 LFTs
or lipid panel tests