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Clinical trial

Safety and Efficacy Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency

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Last updated:1st May 2014

This study will evaluate the safety and efficacy of sebelipase alfa in a broad population of patients with lysosomal acid lipase deficiency (LALD).


Study Type: Interventional
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency
Estimated Enrollment: 30
Study Start Date: May 2014
Estimated Study Completion Date: April 2017
Estimated Primary Completion Date: April 2017 (Final data collection date for primary outcome measure)

Arms:
- Experimental:
sebelipase alfa

Category Value
Study start date 2014-05-01

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