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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02376751
Expanded Access Status : No longer available
First Posted : March 3, 2015
Last Update Posted : June 8, 2016
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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.

Condition or disease Intervention/treatment
Lysosomal Acid Lipase Deficiency Drug: sebelipase alfa

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Study Type : Expanded Access

Intervention Details:
  • Drug: sebelipase alfa
    Other Name: SBC-102, recombinant human lysosomal acid lipase, rhLAL

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

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.

No Contacts or Locations Provided
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Responsible Party: Alexion Pharmaceuticals Identifier: NCT02376751    
Other Study ID Numbers: LAL-EA01
First Posted: March 3, 2015    Key Record Dates
Last Update Posted: June 8, 2016
Last Verified: November 2015
Keywords provided by Alexion Pharmaceuticals:
Cholesteryl Ester Storage Disease
Acid lipase disease
Cholesterol ester hydrolase deficiency
LIPA Deficiency
Wolman disease
Lysosomal Storage Disease
Additional relevant MeSH terms:
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Wolman Disease
Cholesterol Ester Storage Disease
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Infant, Newborn, Diseases
Lipid Metabolism Disorders
Metabolic Diseases