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


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.


Study Type

  • Study Type: Expanded Access


  • Drug: sebelipase alfa

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 8 Months

Maximum Age: N/A

Investigator Details

  • Lead Sponsor
    • Alexion Pharmaceuticals
  • Provider of Information About this Clinical Study
    • Sponsor

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