Ignite Creation Date:
2025-12-25 @ 2:01 AM
Ignite Modification Date:
2025-12-26 @ 12:25 AM
Study NCT ID:
NCT03544060
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2021-09-16
First Post:
2018-05-21
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008072', 'term': 'Hyperlipoproteinemia Type I'}, {'id': 'D006951', 'term': 'Hyperlipoproteinemias'}, {'id': 'C538489', 'term': 'Familial hyperchylomicronemia syndrome'}, {'id': 'D006949', 'term': 'Hyperlipidemias'}, {'id': 'D050171', 'term': 'Dyslipidemias'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000593612', 'term': 'ISIS 304801'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'individual': True}}, 'statusModule': {'overallStatus': 'NO_LONGER_AVAILABLE', 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-09', 'lastUpdateSubmitDate': '2021-09-10', 'studyFirstSubmitDate': '2018-05-21', 'studyFirstSubmitQcDate': '2018-05-21', 'lastUpdatePostDateStruct': {'date': '2021-09-16', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-06-01', 'type': 'ACTUAL'}}, 'conditionsModule': {'keywords': ['Familial Chylomicronemia', 'Familial Lipoprotein Lipase Deficiency', 'Hyperlipoproteinemias', 'Familial Hyperlipoproteinemia Type 1', 'Hyperlipoproteinemia Type 1', 'Hyperchylomicronemia, Familial', 'Lipoprotein Lipase Deficiency, Familial', 'Hyperlipidemias', 'Dyslipidemias', 'Lipid Metabolism Disorders', 'Lipid Metabolism, Inborn Errors', 'Metabolic Diseases', 'Metabolism, Inborn Errors', 'Genetic Diseases, Inborn'], 'conditions': ['Familial Chylomicronemia']}, 'descriptionModule': {'briefSummary': 'The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).', 'detailedDescription': 'The Program is intended to provide expanded access to volanesorsen for eligible patients with FCS who have limited or no available treatment options.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'eligibilityCriteria': "Inclusion Criteria:\n\n* Patients who participated in the APPROACH Open Label trial, must have completed the open label extension trial for volanesorsen for a length of time consistent with the study protocol (≥ 1 year). Patients in the APPROACH open Label trial for less than a year require approval from Akcea to participate in the EAP. Patients who did not participate in the APPROACH open label trial require approval from Akcea for entry into EAP.\n* Patients not participating in the APPROACH open label trial must have a diagnosis of FCS as determined by the participating physician, as outlined in the Volanesorsen EAP protocol. Akcea will review each application to determine eligibility.\n* Male patients and female patients of childbearing potential must continue to use appropriate contraception with their partners, or refrain from sexual activity\n\nExclusion Criteria:\n\n* Patients who have any new conditions or worsening of existing conditions which in the opinion of the Physician would make the patient unsuitable for treatment with volanesorsen.\n* Volanesorsen naïve patients with baseline platelet values ≤ 140,000/mm3\n* Patients not willing to adhere to mandatory blood draws for platelet monitoring\n* Patients who in the opinion of Akcea's medical team, are not eligible candidates for volanesorsen therapy.\n* Any patient who plans to or becomes pregnant.\n* Any patient who was withdrawn from the APPROACH open label study due to a serious adverse event related to volanesorsen therapy."}, 'identificationModule': {'nctId': 'NCT03544060', 'briefTitle': 'Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Akcea Therapeutics'}, 'officialTitle': 'Volanesorsen (ISIS 304801) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)', 'orgStudyIdInfo': {'id': 'ISIS 304801'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Volanesorsen', 'type': 'DRUG', 'otherNames': ['Waylivra'], 'description': 'Volanesorsen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Akcea Therapeutics', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'CaligorRx, Inc.', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'SPONSOR'}}}}