Back to resultsAn Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Primary Purpose
Lysosomal Acid Lipase Deficiency
Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
sebelipase alfa
Sponsored by
About this trial
This is an expanded access trial for Lysosomal Acid Lipase Deficiency focused on measuring Cholesteryl Ester Storage Disease, Acid lipase disease, Cholesterol ester hydrolase deficiency, LIPA Deficiency, Wolman disease, Lysosomal Storage Disease
Eligibility 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.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02376751
Brief Title
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Official Title
AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
Study Type
Expanded Access
2. Study Status
Record Verification Date
November 2015
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lysosomal Acid Lipase Deficiency
Keywords
Cholesteryl Ester Storage Disease, Acid lipase disease, Cholesterol ester hydrolase deficiency, LIPA Deficiency, Wolman disease, Lysosomal Storage Disease
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
sebelipase alfa
Other Intervention Name(s)
SBC-102, recombinant human lysosomal acid lipase, rhLAL
10. Eligibility
Sex
All
Minimum Age & Unit of Time
8 Months
Eligibility 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.
12. IPD Sharing Statement
Learn more about this trial
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
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