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

No longer available
Trial end date:
Target enrollment:
Participant gender:
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.
Lead Sponsor:
Alexion Pharmaceuticals
Inclusion Criteria:

1. Patient is ≥ 8 months of age at commencement of treatment with sebelipase alfa.

2. Patient has a confirmed diagnosis of LAL Deficiency.

3. Patient or patient's parent or legal guardian (if applicable) consents to
participation in the study. If the patient is of minor age, he/she is willing to
provide assent where required per local regulations, and if deemed able to do so.

4. Male and female patients of childbearing potential must use a highly reliable method
of birth control (expected failure rate less than 5% per year) from the time they
commence treatment through 4 weeks after the last dose of sebelipase alfa.

5. Women of childbearing potential must have a negative serum pregnancy test at
commencement of treatment with sebelipase alfa.

Exclusion Criteria:

1. Women who are nursing or pregnant.

2. Patients who received an investigational product within 30 days (for a small molecule)
or 60 days (for a biologic) of commencing treatment, and which in the opinion of the
investigator or Sponsor, may negatively impact patient safety.

3. Patients who have received sebelipase alfa as part of a clinical trial that is
currently active.

4. Patients with known hypersensitivity to eggs.