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

Completed · Phase 2

Conditions studied: Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency

In brief

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.

Key facts

Study ID

NCT01488097

Run by

Alexion Pharmaceuticals, Inc.

People needed

8

Starts

2011-12-12

Expected to finish

2017-06-21

Last updated by the study team

2018-07-20

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• 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

Where it is running

• Study site — Eureka, California, United States
• Study site — Sacramento, California, United States
• Study site — San Francisco, California, United States
• Study site — Minneapolis, Minnesota, United States
• Study site — New York, New York, United States
• Study site — Greater Sudbury, Ontario, Canada
• Study site — Prague, Czechia
• Study site — Paris, France
• Study site — Leeds, United Kingdom
• Study site — Salford, United Kingdom

Full record on ClinicalTrials.gov

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