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An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency Clinical Trial

Official title:
AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY

Clinical Trial Summary

This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid lipase [rhLAL]) until commercial product is available.

Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.

Clinical Trial Description

n/a

Study Design

N/A

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02376751
Study type Expanded Access
Source Alexion Pharmaceuticals
Contact
Status No longer available
Phase N/A
View Details
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