Ignite Creation Date:
2025-12-24 @ 2:14 PM
Ignite Modification Date:
2026-01-02 @ 12:59 AM
Study NCT ID:
NCT00280995
Status:
COMPLETED
Last Update Posted:
2016-08-03
First Post:
2006-01-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006938', 'term': 'Hyperlipoproteinemia Type II'}, {'id': 'D000090542', 'term': 'Homozygous Familial Hypercholesterolemia'}], 'ancestors': [{'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': 'D006951', 'term': 'Hyperlipoproteinemias'}, {'id': 'D006949', 'term': 'Hyperlipidemias'}, {'id': 'D050171', 'term': 'Dyslipidemias'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C524142', 'term': 'mipomersen'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-08', 'completionDateStruct': {'date': '2007-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-08-01', 'studyFirstSubmitDate': '2006-01-20', 'studyFirstSubmitQcDate': '2006-01-20', 'lastUpdatePostDateStruct': {'date': '2016-08-03', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2006-01-24', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2007-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent reduction in LDL-cholesterol from baseline', 'timeFrame': 'Week 7 (Cohorts A-C), Week 15 (Cohort D)'}]}, 'conditionsModule': {'keywords': ['LDL-cholesterol', 'apoB-100', 'Homozygous Familial Hypercholesterolemia'], 'conditions': ['Hypercholesterolemia, Familial']}, 'descriptionModule': {'briefSummary': 'The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 as add-on therapy in subjects with Homozygous Familial Hypercholesterolemia'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Weight ≥ 40 kg for Cohorts A, B, \\& C; Weight ≥ 50 kg for Cohort D\n* Diagnosis of Homozygous Familial Hypercholesterolemia.\n* Female must be non-pregnant and non-lactating.\n* On stable lipid lowering therapy for at least 4 weeks.\n* Lipid values that meet the pre-specified criteria.\n\nExclusion Criteria:\n\n* Subject had heart problems in the prior 6 months.\n* Subject has elevated ALT, AST, or CPK.\n* History of renal disease, liver disease, or malignancy.\n* Use of oral anticoagulants, unless the dose has been stable for 4 weeks\n* Have any other conditions, which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the subject participating in or completing the study.'}, 'identificationModule': {'nctId': 'NCT00280995', 'briefTitle': 'Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Kastle Therapeutics, LLC'}, 'officialTitle': 'A Phase 2, Open-Label, Dose Escalation Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects', 'orgStudyIdInfo': {'id': '301012CS8'}, 'secondaryIdInfos': [{'id': 'EudraCT No.: 2005-004796-38'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort A', 'description': 'Loading doses followed by weekly maintenance doses', 'interventionNames': ['Drug: ISIS 301012']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort B', 'description': 'Loading doses followed by weekly maintenance doses', 'interventionNames': ['Drug: ISIS 301012']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort C', 'description': 'Loading doses followed by weekly maintenance doses', 'interventionNames': ['Drug: ISIS 301012']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort D', 'description': 'Loading doses followed by extended weekly maintenance doses', 'interventionNames': ['Drug: ISIS 301012']}], 'interventions': [{'name': 'ISIS 301012', 'type': 'DRUG', 'description': '100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36', 'armGroupLabels': ['Cohort B']}, {'name': 'ISIS 301012', 'type': 'DRUG', 'description': '200 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36', 'armGroupLabels': ['Cohort C']}, {'name': 'ISIS 301012', 'type': 'DRUG', 'description': '300 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, 36, 43, 50, 57, 64,71, 78, and 85', 'armGroupLabels': ['Cohort D']}, {'name': 'ISIS 301012', 'type': 'DRUG', 'description': '50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36', 'armGroupLabels': ['Cohort A']}]}, 'contactsLocationsModule': {'locations': [{'zip': '60610', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '10021', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27103', 'city': 'Winston-Salem', 'state': 'North Carolina', 'country': 'United States', 'geoPoint': {'lat': 36.09986, 'lon': -80.24422}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '1105 AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}], 'overallOfficials': [{'name': 'Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Genzyme, a Sanofi Company'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Kastle Therapeutics, LLC', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Ionis Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}], 'responsibleParty': {'oldNameTitle': 'Medical Monitor', 'oldOrganization': 'Genzyme Coporation'}}}}