Ignite Creation Date:
2025-12-24 @ 11:55 PM
Ignite Modification Date:
2026-03-01 @ 1:45 PM
Study NCT ID:
NCT02376751
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2016-06-08
First Post:
2015-02-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Sponsor:
Alexion Pharmaceuticals, Inc.
Organization:
Study Overview
Official Title:
AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2015-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: