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

Expanded access is no longer available for this treatment.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02376751
First Posted: March 3, 2015
Last Update Posted: June 8, 2016
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.
Information provided by (Responsible Party):
Alexion Pharmaceuticals
  Purpose

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 Intervention
Lysosomal Acid Lipase Deficiency Drug: sebelipase alfa

Study Type: Expanded Access     What is Expanded Access?
Official Title: AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

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

Information from the National Library of Medicine

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

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.
  Contacts and Locations
No Contacts or Locations Provided
  More Information

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02376751     History of Changes
Other Study ID Numbers: LAL-EA01
First Submitted: February 25, 2015
First Posted: March 3, 2015
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:
Wolman Disease
Cholesterol Ester Storage Disease
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Infant, Newborn, Diseases
Lipid Metabolism Disorders
Metabolic Diseases