Ignite Creation Date:
2025-12-25 @ 12:10 AM
Ignite Modification Date:
2026-02-18 @ 12:20 PM
Study NCT ID:
NCT02173158
Status:
COMPLETED
Last Update Posted:
2018-10-10
First Post:
2014-06-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000090542', 'term': 'Homozygous Familial Hypercholesterolemia'}], 'ancestors': [{'id': 'D006938', 'term': 'Hyperlipoproteinemia Type II'}, {'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': 'C473731', 'term': 'BMS201038'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'alison.long@aegerion.com', 'phone': '857-242-5142', 'title': 'Alison Long, MD - VP Clinical', 'organization': 'Aegerion Pharmaceuticals, Inc.'}, 'certainAgreement': {'otherDetails': 'Described in site contract', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Efficacy Phase', 'description': 'Baseline to Week 26', 'otherNumAtRisk': 9, 'deathsNumAtRisk': 9, 'otherNumAffected': 9, 'seriousNumAtRisk': 9, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Safety Phase', 'description': 'Week 26 to Week 56', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 7, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Abdominal pain lower', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, 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{'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Periodontitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Device breakage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Feeling abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, 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'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Subcutaneous haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Tooth fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Iron deficiency anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Liver function test abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'White blood cell count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Mylagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Drug eruption', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Pruritus allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 9, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 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safety phase and had at least one assessment during the safety phase'}, {'type': 'SECONDARY', 'title': 'Change in Non-HDL-C', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Lomitapide', 'description': 'Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.'}], 'classes': [{'categories': [{'measurements': [{'value': '-34.6', 'spread': '22.50', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline', 'unitOfMeasure': 'Percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full analysis set for the safety phase included all subjects who received study drug during the safety phase and had at least one assessment during the safety phase'}, {'type': 'SECONDARY', 'title': 'Change in VLDL-C', 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'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 9}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-04-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-02', 'completionDateStruct': {'date': '2015-12-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-02-21', 'studyFirstSubmitDate': '2014-06-23', 'resultsFirstSubmitDate': '2018-02-09', 'studyFirstSubmitQcDate': '2014-06-23', 'lastUpdatePostDateStruct': {'date': '2018-10-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-02-21', 'studyFirstPostDateStruct': {'date': '2014-06-24', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-10-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2015-04-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent Change in LDL-C', 'timeFrame': 'Baseline to Week 26', 'description': 'Mean percent change from baseline'}], 'secondaryOutcomes': [{'measure': 'Change in Total Cholesterol', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in Apo B', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in Triglycerides', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in Non-HDL-C', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in VLDL-C', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in Lp(a)', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in HDL-C', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in Apo AI', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}, {'measure': 'Change in LDL-C', 'timeFrame': 'Baseline to Week 56', 'description': 'Mean percent change from baseline'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Familial Hypercholesterolemia - Homozygous']}, 'descriptionModule': {'briefSummary': 'Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy.', 'detailedDescription': 'This is a Phase 3, single-arm, open-label, multicenter clinical trial to evaluate both the efficacy and long-term safety of lomitapide in Japanese patients with HoFH receiving maximally-tolerated, stable lipid-lowering therapy. This study is comprised of a run-in period, a primary 26-week Efficacy Phase, and a 30-week Safety Phase.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy\n2. Diagnosis of functional HoFH\n3. Body weight ≥ 40 kg and \\< 136 kg\n4. Negative pregnancy test at screening\n\nExclusion Criteria:\n\n1. Uncontrolled hypertension\n2. History of chronic renal insufficiency\n3. History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening\n4. Any major surgical procedure occurring \\< 3 months prior to the screening visit\n5. Cardiac insufficiency\n6. Previous organ transplantation\n7. History of a non-skin malignancy within the previous 3 years\n8. Patients who are not able to limit their alcohol intake\n9. Participation in an investigational drug study within 6 weeks prior to the screening visit\n10. Known significant gastrointestinal bowel disease\n11. Nursing mothers\n12. Serious or unstable medical or psychological conditions\n13. Requirement for certain prohibited medications known to be potentially hepatotoxic\n14. Use of strong or moderate inhibitors of CYP3A4\n15. Use of simvastatin at doses \\>10 mg per day\n16. Documented diagnosis of any liver disease'}, 'identificationModule': {'nctId': 'NCT02173158', 'briefTitle': 'Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Aegerion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 3, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy', 'orgStudyIdInfo': {'id': 'AEGR-733-030'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'lomitapide', 'description': 'Maximum tolerated dose of lomitapide (up to 60mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.', 'interventionNames': ['Drug: lomitapide']}], 'interventions': [{'name': 'lomitapide', 'type': 'DRUG', 'otherNames': ['Juxtapid, Lojuxta'], 'armGroupLabels': ['lomitapide']}]}, 'contactsLocationsModule': {'locations': [{'zip': '830-8522', 'city': 'Kurume-shi', 'state': 'Fukuoka', 'country': 'Japan'}, {'zip': '920-8641', 'city': 'Kanazawa', 'state': 'Ishikawa-ken', 'country': 'Japan', 'geoPoint': {'lat': 36.6, 'lon': 136.61667}}, {'zip': '530-00001', 'city': 'Osakashi', 'state': 'Osaka', 'country': 'Japan'}, {'zip': '565-8565', 'city': 'Suita-shi', 'state': 'Osaka', 'country': 'Japan'}, {'zip': '359-1142', 'city': 'Tokorozawa', 'state': 'Saitama', 'country': 'Japan', 'geoPoint': {'lat': 35.79916, 'lon': 139.46903}}, {'zip': '113-8519', 'city': 'Bunkyo-ku', 'state': 'Tokyo', 'country': 'Japan'}], 'overallOfficials': [{'name': 'Mariko Harada-Shiba, M.D., Ph.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'National Cerebral and Cardiovascular Center Research Institute'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Aegerion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}