Clinical Trials Directory

Trials / No Longer Available

No Longer AvailableNCT02376751

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

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

Status
No Longer Available
Phase
Study type
Expanded Access
Enrollment
Sponsor
Alexion Pharmaceuticals, Inc. · Industry
Sex
All
Age
8 Months
Healthy volunteers

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.

Conditions

Interventions

TypeNameDescription
DRUGsebelipase alfa

Timeline

First posted
2015-03-03
Last updated
2016-06-08

Source: ClinicalTrials.gov record NCT02376751. Inclusion in this directory is not an endorsement.