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

Completed · Phase 1/Phase 2

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

In brief

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.

Key facts

Study ID: NCT01307098
Run by: Alexion Pharmaceuticals, Inc.
People needed: 9
Starts: 2011-04-25
Expected to finish: 2012-01-06
Last updated by the study team: 2018-12-11

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Male or female participants ≥ 18 and ≤ 65 years of age
Documented decreased LAL activity
Evidence of liver involvement

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 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

Where it is running

Study site — Stanford, California, United States
Study site — New York, New York, United States
Study site — Pittsburgh, Pennsylvania, United States
Study site — Prague, Czechia
Study site — Paris, France
Study site — Cambridge, United Kingdom
Study site — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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