Ignite Creation Date:
2025-12-24 @ 11:55 PM
Ignite Modification Date:
2026-03-01 @ 1:45 PM
Study NCT ID:
NCT02376751
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2016-06-08
First Post:
2015-02-25
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015223', 'term': 'Wolman Disease'}, {'id': 'D015217', 'term': 'Cholesterol Ester Storage Disease'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}], 'ancestors': [{'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000603932', 'term': 'Sebelipase alfa'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS'}, 'statusModule': {'overallStatus': 'NO_LONGER_AVAILABLE', 'statusVerifiedDate': '2015-11', 'lastUpdateSubmitDate': '2016-06-06', 'studyFirstSubmitDate': '2015-02-25', 'studyFirstSubmitQcDate': '2015-03-02', 'lastUpdatePostDateStruct': {'date': '2016-06-08', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2015-03-03', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['Cholesteryl Ester Storage Disease', 'Acid lipase disease', 'Cholesterol ester hydrolase deficiency', 'LIPA Deficiency', 'Wolman disease', 'Lysosomal Storage Disease'], 'conditions': ['Lysosomal Acid Lipase Deficiency']}, 'descriptionModule': {'briefSummary': '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.\n\nPatients enrolled in the expanded access protocol will receive 1 mg/kg intravenous infusions of sebelipase alfa every other week.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '8 Months', 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Patient is ≥ 8 months of age at commencement of treatment with sebelipase alfa.\n2. Patient has a confirmed diagnosis of LAL Deficiency.\n3. 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.\n4. 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.\n5. Women of childbearing potential must have a negative serum pregnancy test at commencement of treatment with sebelipase alfa.\n\nExclusion Criteria:\n\n1. Women who are nursing or pregnant.\n2. 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.\n3. Patients who have received sebelipase alfa as part of a clinical trial that is currently active.\n4. Patients with known hypersensitivity to eggs."}, 'identificationModule': {'nctId': 'NCT02376751', 'briefTitle': 'An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY', 'orgStudyIdInfo': {'id': 'LAL-EA01'}}, 'armsInterventionsModule': {'interventions': [{'name': 'sebelipase alfa', 'type': 'DRUG', 'otherNames': ['SBC-102, recombinant human lysosomal acid lipase, rhLAL']}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}