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

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Last updated:1st Nov 2011

This is an extension study to the first clinical study of SBC-102 (sebelipase alfa) for the treatment of LAL Deficiency. It is an open label study in adult patients with liver dysfunction due to Lysosomal Acid Lipase (LAL) Deficiency. This study will assess the long-term safety, tolerability, and efficacy of SBC-102. The targeted number for this study is 9 evaluable subjects.


Study Type: Interventional
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Multicenter Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 in Adult Subjects With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency Who Previously Received Treatment in Study LAL-CL01
Enrollment: 8
Study Start Date: November 2011
Estimated Study Completion Date: June 2017
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)

Arms:
- Experimental:
Cohort 1
- Experimental: Cohort 2
- Experimental: Cohort 3


Related journal: Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease.

Category Value
Study start date 2011-11-01

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