An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
NCT ID: NCT02376751
Last Updated: 2016-06-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NO_LONGER_AVAILABLE
EXPANDED_ACCESS
Brief Summary
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Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.
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Detailed Description
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Conditions
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Interventions
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sebelipase alfa
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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.
8 Months
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Other Identifiers
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LAL-EA01
Identifier Type: -
Identifier Source: org_study_id
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