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.
Patients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.
Brief Title
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Conditions
Lysosomal Acid Lipase Deficiency
Eligibility Criteria
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.
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.
Inclusion Criteria
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.
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.
Gender
All
Gender Based
false
Keywords
Cholesteryl Ester Storage Disease
Acid lipase disease
Cholesterol ester hydrolase deficiency
LIPA Deficiency
Wolman disease
Lysosomal Storage Disease
Last Update Post Date
Last Update Post Date Type
Estimated
Last Update Submit Date
Minimum Age
8 Months
NCT Id
NCT02376751
Org Class
Industry
Org Full Name
Alexion Pharmaceuticals, Inc.
Org Study Id
LAL-EA01
Overall Status
No longer available
Official Title
AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
Allan Wolkoff
Investigator Email
allan.wolkoff@einsteinmed.org
Investigator Phone
718-430-3798