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

NCT02376751 | Unknown

• 1 other identifier: LAL-EA01
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Lysosomal Acid Lipase Deficiency

Keywords

Cholesteryl Ester Storage Disease; Acid lipase disease; Cholesterol ester hydrolase deficiency; LIPA Deficiency; Wolman disease; Lysosomal Storage Disease

Interventions

sebelipase alfa DRUG

Also known as: SBC-102, recombinant human lysosomal acid lipase, rhLAL

Eligibility Criteria

• Age: 8 Months+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient is ≥ 8 months of age at commencement of treatment with sebelipase alfa.
• Patient has a confirmed diagnosis of LAL Deficiency.
• 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.
• 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.
• Women of childbearing potential must have a negative serum pregnancy test at commencement of treatment with sebelipase alfa.

You may not qualify if:

• Women who are nursing or pregnant.
• 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.
• Patients who have received sebelipase alfa as part of a clinical trial that is currently active.
• Patients with known hypersensitivity to eggs.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Wolman Disease; Cholesterol Ester Storage Disease; Lysosomal Storage Diseases

Interventions

Sebelipase alfa

Condition Hierarchy (Ancestors)

Lipidoses; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 25, 2015
• First Posted: March 3, 2015
• Last Updated: June 8, 2016

Record last verified: 2015-11