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

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 Overview

• Status: No longer available
• Conditions: Lysosomal Acid Lipase Deficiency
• Intervention / Treatment: Drug: sebelipase alfa

• Study Type: Expanded Access

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals

Study record dates

Study Registration Dates

• First Submitted: February 25, 2015
• First Submitted That Met QC Criteria: March 2, 2015
• First Posted (Estimate): March 3, 2015

Study Record Updates

• Last Update Posted (Estimate): June 8, 2016
• Last Update Submitted That Met QC Criteria: June 6, 2016
• Last Verified: November 1, 2015

More Information

Terms related to this study

• Keywords: Lysosomal Storage Disease; Acid lipase disease; Cholesterol ester hydrolase deficiency; LIPA Deficiency; Wolman disease; Cholesteryl Ester Storage Disease
• Additional Relevant MeSH Terms: Metabolic Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Lipidoses; Lipid Metabolism, Inborn Errors; Cholesterol Ester Storage Disease; Wolman Disease
• Other Study ID Numbers: LAL-EA01