An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
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|ClinicalTrials.gov Identifier: NCT02376751|
Recruitment Status : No longer available
First Posted : March 3, 2015
Last Update Posted : June 8, 2016
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.
|Condition or disease||Intervention/treatment|
|Lysosomal Acid Lipase Deficiency||Drug: sebelipase alfa|
|Study Type :||Expanded Access|
|Official Title:||AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY|
Drug: sebelipase alfa