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

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

9

Participants

Timeline

Start Date

April 25, 2011

Primary Completion Date

January 6, 2012

Study Completion Date

January 6, 2012

Conditions
Cholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency
Interventions
DRUG

Sebelipase alfa 0.35 mg/kg

Sebelipase alfa is a recombinant human lysosomal acid lipase.

DRUG

Sebelipase alfa 1 mg/kg

Sebelipase alfa is a recombinant human lysosomal acid lipase.

DRUG

Sebelipase alfa 3 mg/kg

Sebelipase alfa is a recombinant human lysosomal acid lipase.

Trial Locations (7)

10029

New York

12000

Prague

15213

Pittsburgh

75743

Paris

94305

Stanford

CB20QQ

Cambridge

M139WL

Manchester

Sponsors
All Listed Sponsors
lead

Alexion Pharmaceuticals, Inc.

INDUSTRY