Lysosomal Acid Lipase Deficiency Clinical Trial
Official title:
AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY
NCT number | NCT02376751 |
Other study ID # | LAL-EA01 |
Secondary ID | |
Status | No longer available |
Phase | N/A |
First received | February 25, 2015 |
Last updated | June 6, 2016 |
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 |
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.
Status | No longer available |
Enrollment | 0 |
Est. completion date | |
Est. primary completion date | |
Accepts healthy volunteers | |
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. |
N/A
Country | Name | City | State |
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n/a |
Lead Sponsor | Collaborator |
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Alexion Pharmaceuticals |
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