An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Status:
No longer available
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
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.