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

Lysosomal Acid Lipase Deficiency Clinical Trial

Official title:

AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT02376751
• Other study ID #: LAL-EA01
• Status: No longer available
• Phase: N/A
• First received: February 25, 2015
• Last updated: June 6, 2016

Study information

• Verified date: November 2015
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: Both
• Age group: 8 Months and older

Eligibility

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 Design

N/A

Related Conditions & MeSH terms

• Lysosomal Acid Lipase Deficiency
• Wolman Disease

Intervention

Drug:
• sebelipase alfa

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals