An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Study Identifier:
LAL-EA01
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: sebelipase alfa
Date
N/A - N/A
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 8+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Sex
Female & Male
Age
8+ years
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: sebelipase alfa
Date
N/A - N/A
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 8+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Protocol 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.
Trial Locations
No locations found.