Ignite Creation Date:
2025-12-25 @ 5:04 AM
Ignite Modification Date:
2025-12-26 @ 4:06 AM
Study NCT ID:
NCT04233918
Status:
COMPLETED
Last Update Posted:
2024-07-16
First Post:
2020-01-06
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Sponsor:
Organization:
Raw JSON
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The investigator must provide the sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up)', 'description': 'Part A - up to week 24\n\nPart B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock.\n\nPart C - up to a 48-week treatment period and 24-week follow-up', 'eventGroups': [{'id': 'EG000', 'title': 'Part A Evinacumab 15mg', 'description': 'Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 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'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 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'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 10, 'numAffected': 10}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Gastrointestinal viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Oral herpes', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 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'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 2, 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14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Vitamin D decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Iron deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, 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'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Miliaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Poikilocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Conjunctivitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Papilloedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Retinal thickening', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Apheresis related complication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Device malfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Product Issues', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Gastritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Infusion site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Lipoprotein (a) increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Burn oral cavity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Limb injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Sunburn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Thermal burn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Osteochondrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}], 'seriousEvents': [{'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Aortic valve stenosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: Evinacumab 15mg/Kg IV', 'description': 'Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.'}], 'classes': [{'categories': [{'measurements': [{'value': '238', 'spread': '90.8', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At day 12', 'description': 'Cmax was obtained directly from the plasma concentration versus time curve.', 'unitOfMeasure': 'Milligrams per Liter (mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Pharmacokinetic (PK) Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: Evinacumab 15mg/Kg IV', 'description': 'Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.'}], 'classes': [{'categories': [{'measurements': [{'value': '4576', 'spread': '1568', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Up to Week 12', 'description': 'AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.', 'unitOfMeasure': 'Days*Milligrams per Liter (day*mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part A: Terminal Half-Life (t1/2) of Evinacumab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: Evinacumab 15mg/Kg IV', 'description': 'Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.'}], 'classes': [{'categories': [{'measurements': [{'value': '7.69', 'groupId': 'OG000', 'lowerLimit': '6.18', 'upperLimit': '12.4'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Up to week 12', 'description': 'T1/2 was defined as the time required for the plasma concentration of drug to decrease 50 percent in the final stage of its elimination.', 'unitOfMeasure': 'Days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '-48.3', 'groupId': 'OG000', 'lowerLimit': '-68.8', 'upperLimit': '-27.8'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change was calculated as 100 multiplied by (calculated LDL-C value at Week 24 minus calculated LDL-C value at baseline) divided by calculated LDL-C value at baseline.', 'unitOfMeasure': 'Percentage of Change', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The intent-to-treat (ITT) population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}, {'type': 'SECONDARY', 'title': 'Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: Evinacumab 15mg/Kg IV', 'description': 'Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.'}, {'id': 'OG001', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Part A: up to Week 24; Part B: up to Week 48', 'description': 'Any untoward medical occurrence in a participant who received investigational medicinal product (IMP) was considered an AE without regard to possibility of causal relationship with this treatment. A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAE was defined as AE starting/worsening after first intake of study drug. TEAE included participants with both serious and non-serious AEs.\n\n1 participant experienced an AE during part B that was recorded after Part B database lock. This was not reflected in the reported endpoint number of participants of 10.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set (SAF) includes all patients in Part A or Part B who received at least 1 dose or part of a dose of study drug in the respective study treatment period.'}, {'type': 'SECONDARY', 'title': 'Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '-41.3', 'groupId': 'OG000', 'lowerLimit': '-58.9', 'upperLimit': '-23.8'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in Apo B from baseline to Week 24 was reported.', 'unitOfMeasure': 'Percentage of Change', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}, {'type': 'SECONDARY', 'title': 'Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '-48.9', 'groupId': 'OG000', 'lowerLimit': '-68.1', 'upperLimit': '-29.7'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in Non-HDL-C from baseline to Week 24 was reported.', 'unitOfMeasure': 'Percentage of Change', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}, {'type': 'SECONDARY', 'title': 'Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '-49.1', 'groupId': 'OG000', 'lowerLimit': '-64.9', 'upperLimit': '-33.2'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in TC from baseline to Week 24 was reported.', 'unitOfMeasure': 'Percentage of Change', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}, {'type': 'SECONDARY', 'title': 'Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '78.6', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Week 24', 'description': 'Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 was reported.', 'unitOfMeasure': 'Percentage of Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}, {'type': 'SECONDARY', 'title': 'Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have Negative/Negative and Null/Null Mutations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'title': 'Negative/negative mutations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-67.7', 'spread': '6.5', 'groupId': 'OG000'}]}]}, {'title': 'Null/Null mutations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-57.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24', 'description': 'Participants with HoFH was classified based on the phenotype of the Low-density lipoprotein receptor (LDLR) mutation(s), ranging from defective mutations (where the LDLR retains some LDL-binding functionality) to null or negative mutations where no functioning LDLR was expressed. Participants who have LDLR activity \\<15% are considered null and participants whose LDLR activity was impaired but \\>15% are LDLR defective. Percent change in calculated LDL-C from baseline to Week 24 in participants who have negative/negative and null/null mutations was reported.', 'unitOfMeasure': 'Percentage of Change', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number Analyzed" signifies those participants who were evaluable at specified category.'}, {'type': 'SECONDARY', 'title': 'Part B: Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '-37.3', 'groupId': 'OG000', 'lowerLimit': '-42.2', 'upperLimit': '-32.3'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in Lp(a) from baseline to Week 24 was reported.', 'unitOfMeasure': 'Percentage of Change', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}, {'type': 'SECONDARY', 'title': 'Part B: Absolute Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '-131.9', 'spread': '30.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Absolute change in LDL-C from baseline at Week 24 was reported', 'unitOfMeasure': 'Milligrams per Deciliter (mg/dL)', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}, {'type': 'SECONDARY', 'title': 'Part B: Serum Concentration of Total Evinacumab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'title': 'Week 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'spread': '0', 'groupId': 'OG000'}]}]}, {'title': 'Week 0.006', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '256', 'spread': '58.0', 'groupId': 'OG000'}]}]}, {'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '62.6', 'spread': '22.6', 'groupId': 'OG000'}]}]}, {'title': 'Week 4.006', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '293', 'spread': '92.3', 'groupId': 'OG000'}]}]}, {'title': 'Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '98.8', 'spread': '37.7', 'groupId': 'OG000'}]}]}, {'title': 'Week 8.006', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '356', 'spread': '76', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '120', 'spread': '46.5', 'groupId': 'OG000'}]}]}, {'title': 'Week 12.006', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '363', 'spread': '82.1', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '140', 'spread': '92.5', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-dose at Weeks 0, 4, 8, 12; End of infusion at Weeks 0.006, 4.006, 8.006, 12.006 and 24', 'description': 'Serum concentration of total evinacumab was reported. Pre-dose samples at week 0 were assayed and the reported value is based on actual measurement.', 'unitOfMeasure': 'Milligrams per Liter (mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug. Here, "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified timepoints.'}, {'type': 'SECONDARY', 'title': 'Part B: Maximum Serum Concentration at Steady State (Cmax,ss) of Evinacumab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '428.9', 'spread': '113.7', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Post-dose up to day 169', 'description': 'Maximum serum concentration (Cmax,ss) steady state following drug administration.', 'unitOfMeasure': 'Milligrams per Liter (mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Part B: Area Under the Serum Concentration-time Curve at Steady State (AUCtau.ss) of Evinacumab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '7019', 'spread': '2561', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Post-dose up to day 169', 'description': 'AUCtau.ss was defined as area under the serum concentration-time curve at steady state of evinacumab', 'unitOfMeasure': 'Day*Milligrams per Liter (Day*mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Part B: Minimum Serum Concentration at Steady State (Ctrough.ss) of Evinacumab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'categories': [{'measurements': [{'value': '171.8', 'spread': '79.5', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Post-dose up to day 169', 'description': 'Ctrough.ss was defined as minimum serum concentration at steady state of evinacumab', 'unitOfMeasure': 'Milligrams per Liter (mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have by Null/Null vs. Non-null/Null and Negative/Negative vs.Non-negative/Negative Mutations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}], 'classes': [{'title': 'Negative/Negative', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-67.7', 'spread': '6.5', 'groupId': 'OG000'}]}]}, {'title': 'Non-Negative/Negative', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-43.0', 'spread': '12.8', 'groupId': 'OG000'}]}]}, {'title': 'Null/Null', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-57.2', 'spread': 'NA', 'comment': 'Standard Error cannot be calculated with only 1 participant', 'groupId': 'OG000'}]}]}, {'title': 'Non-Null/Null', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-47.6', 'spread': '11.3', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24', 'unitOfMeasure': 'Percentage of Change', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Part A: Evinacumab 15mg/Kg IV', 'description': 'In Part A, Participants received single IV infusion of evinacumab at a dose of 15 mg/kg on Day 1.'}, {'id': 'FG001', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'In Part B, Participants received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 24.'}, {'id': 'FG002', 'title': 'Part A to C', 'description': 'All participants who completed Part A received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 48 in Part C.'}, {'id': 'FG003', 'title': 'Part B to C', 'description': 'All participants who completed Part B received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 48 in Part C.'}], 'periods': [{'title': 'Period 1: Part A and B', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '14'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '14'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}, {'title': 'Part C (Extension Period)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '6'}, {'groupId': 'FG003', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '6'}, {'groupId': 'FG003', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'A total of 23 participants were screened to Part A and Part B. 6 participants were enrolled in Part A, 14 participants in Part B. 3 participants were considered screen failures. 2 withdrew consent, 1 due to Other. Participants who enrolled in Part A of study were not eligible to participate in Part B. All 20 participants completed part C.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Part A: Evinacumab 15mg/Kg IV', 'description': 'Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.'}, {'id': 'BG001', 'title': 'Part B: Evinacumab 15mg/Kg IV Q4W', 'description': 'Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '8.8', 'spread': '1.72', 'groupId': 'BG000'}, {'value': '9.1', 'spread': '1.94', 'groupId': 'BG001'}, {'value': '9', 'spread': '1.84', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-05-25', 'size': 878314, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_002.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-05-13T13:14', 'hasProtocol': True}, {'date': '2021-02-18', 'size': 960751, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-04-19T00:56', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 20}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-06-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-06', 'dispFirstSubmitDate': '2023-01-30', 'completionDateStruct': {'date': '2023-05-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-06-25', 'studyFirstSubmitDate': '2020-01-06', 'resultsFirstSubmitDate': '2023-04-19', 'studyFirstSubmitQcDate': '2020-01-16', 'dispFirstPostDateStruct': {'date': '2023-06-07', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2024-07-16', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-06-05', 'studyFirstPostDateStruct': {'date': '2020-01-18', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-06-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-01-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab', 'timeFrame': 'At day 12', 'description': 'Cmax was obtained directly from the plasma concentration versus time curve.'}, {'measure': 'Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab', 'timeFrame': 'Up to Week 12', 'description': 'AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.'}, {'measure': 'Part A: Terminal Half-Life (t1/2) of Evinacumab', 'timeFrame': 'Up to week 12', 'description': 'T1/2 was defined as the time required for the plasma concentration of drug to decrease 50 percent in the final stage of its elimination.'}, {'measure': 'Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change was calculated as 100 multiplied by (calculated LDL-C value at Week 24 minus calculated LDL-C value at baseline) divided by calculated LDL-C value at baseline.'}], 'secondaryOutcomes': [{'measure': 'Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'timeFrame': 'Part A: up to Week 24; Part B: up to Week 48', 'description': 'Any untoward medical occurrence in a participant who received investigational medicinal product (IMP) was considered an AE without regard to possibility of causal relationship with this treatment. A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAE was defined as AE starting/worsening after first intake of study drug. TEAE included participants with both serious and non-serious AEs.\n\n1 participant experienced an AE during part B that was recorded after Part B database lock. This was not reflected in the reported endpoint number of participants of 10.'}, {'measure': 'Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in Apo B from baseline to Week 24 was reported.'}, {'measure': 'Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in Non-HDL-C from baseline to Week 24 was reported.'}, {'measure': 'Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in TC from baseline to Week 24 was reported.'}, {'measure': 'Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24', 'timeFrame': 'Week 24', 'description': 'Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 was reported.'}, {'measure': 'Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have Negative/Negative and Null/Null Mutations', 'timeFrame': 'Baseline to Week 24', 'description': 'Participants with HoFH was classified based on the phenotype of the Low-density lipoprotein receptor (LDLR) mutation(s), ranging from defective mutations (where the LDLR retains some LDL-binding functionality) to null or negative mutations where no functioning LDLR was expressed. Participants who have LDLR activity \\<15% are considered null and participants whose LDLR activity was impaired but \\>15% are LDLR defective. Percent change in calculated LDL-C from baseline to Week 24 in participants who have negative/negative and null/null mutations was reported.'}, {'measure': 'Part B: Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24', 'timeFrame': 'Baseline to Week 24', 'description': 'Percent change in Lp(a) from baseline to Week 24 was reported.'}, {'measure': 'Part B: Absolute Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline at Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Absolute change in LDL-C from baseline at Week 24 was reported'}, {'measure': 'Part B: Serum Concentration of Total Evinacumab', 'timeFrame': 'Pre-dose at Weeks 0, 4, 8, 12; End of infusion at Weeks 0.006, 4.006, 8.006, 12.006 and 24', 'description': 'Serum concentration of total evinacumab was reported. Pre-dose samples at week 0 were assayed and the reported value is based on actual measurement.'}, {'measure': 'Part B: Maximum Serum Concentration at Steady State (Cmax,ss) of Evinacumab', 'timeFrame': 'Post-dose up to day 169', 'description': 'Maximum serum concentration (Cmax,ss) steady state following drug administration.'}, {'measure': 'Part B: Area Under the Serum Concentration-time Curve at Steady State (AUCtau.ss) of Evinacumab', 'timeFrame': 'Post-dose up to day 169', 'description': 'AUCtau.ss was defined as area under the serum concentration-time curve at steady state of evinacumab'}, {'measure': 'Part B: Minimum Serum Concentration at Steady State (Ctrough.ss) of Evinacumab', 'timeFrame': 'Post-dose up to day 169', 'description': 'Ctrough.ss was defined as minimum serum concentration at steady state of evinacumab'}, {'measure': 'Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have by Null/Null vs. Non-null/Null and Negative/Negative vs.Non-negative/Negative Mutations', 'timeFrame': 'Baseline to Week 24'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['HoFH'], 'conditions': ['Homozygous Familial Hypercholesterolemia']}, 'referencesModule': {'references': [{'pmid': '37860863', 'type': 'DERIVED', 'citation': 'Wiegman A, Greber-Platzer S, Ali S, Reijman MD, Brinton EA, Charng MJ, Srinivasan S, Baker-Smith C, Baum S, Brothers JA, Hartz J, Moriarty PM, Mendell J, Bihorel S, Banerjee P, George RT, Hirshberg B, Pordy R. Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia. Circulation. 2024 Jan 30;149(5):343-353. doi: 10.1161/CIRCULATIONAHA.123.065529. Epub 2023 Oct 20.'}]}, 'descriptionModule': {'briefSummary': 'The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).\n\nThe primary objective for Part B of the study is to demonstrate a reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab in pediatric (5 to 11 years of age) patients with HoFH.\n\nThe secondary objective for Part A of the study is to evaluate the safety and tolerability of evinacumab administered intravenous (IV) in pediatric patients with HoFH.\n\nThe secondary objectives for Part B of the study are:\n\n* To evaluate the effect of evinacumab on other lipid parameters (ie, apolipoprotein B (Apo B), non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC), lipoprotein a \\[Lp(a)\\]) in pediatric patients with HoFH\n* To evaluate the safety and tolerability of evinacumab administered IV in pediatric patients with HoFH\n* To assess the PK of evinacumab in pediatric patients with HoFH\n* To assess the immunogenicity of evinacumab in pediatric patients with HoFH over time\n* To evaluate patient efficacy by mutation status', 'detailedDescription': 'Part A is Phase 1b Part B is Phase 3'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Diagnosis of functional HoFH by either genetic or clinical criteria as defined in the protocol\n2. LDL-C \\>130 mg/dL at the screening visit\n3. Body weight ≥15 kg\n4. Receiving stable maximally tolerated therapy\\*at the screening visit \\*Maximally tolerated therapy could include a daily statin.\n5. Willing and able to comply with clinic visits and study-related procedures\n6. Parent(s) or legal guardian(s) must provide the signed informed consent form (ICF). Patients ≥5 years of age (or above age determined by the IRB/EC and in accordance with the local regulations and requirements) must also provide informed assent forms (IAFs) to enroll in the study, and sign and date a separate IAF or ICF signed by the parent(s)/legal guardian(s) (as appropriate based on local regulations and requirements)\n\nKey Exclusion Criteria:\n\n1. Background pharmacologic LMT, nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/regimen that has not been stable for at least 4 weeks (8 weeks for PCSK9 inhibitors) before the screening visit and patient is unwilling to enter the run-in period\n2. For patients entering Part A, unable to temporarily discontinue apheresis from the baseline visit through the week 4 visit\n3. Receiving lipid apheresis, a setting (if applicable) and schedule that has not been stable for approximately 8 weeks before the screening visit or an apheresis schedule that is not anticipated to be stable over the duration of the treatment period (48 weeks).\n4. Plasmapheresis within 8 weeks of the screening visit, or plans to undergo plasmapheresis during Part A or Part B\n5. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins\n6. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled diabetes as defined in the protocol\n\nNote: Other protocol-defined criteria apply'}, 'identificationModule': {'nctId': 'NCT04233918', 'briefTitle': 'Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia', 'organization': {'class': 'INDUSTRY', 'fullName': 'Regeneron Pharmaceuticals'}, 'officialTitle': 'A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia', 'orgStudyIdInfo': {'id': 'R1500-CL-17100'}, 'secondaryIdInfos': [{'id': '2019-001931-30', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Evinacumab', 'description': 'Part A: Single intravenous (IV) dose Part B: IV dose every 4 weeks (Q4W) until week 20 Part C: IV dose Q4W', 'interventionNames': ['Drug: Evinacumab']}], 'interventions': [{'name': 'Evinacumab', 'type': 'DRUG', 'otherNames': ['REGN1500', 'Evkeeza™'], 'description': 'Part A: Single IV dose Part B \\& C: IV dose Q4W', 'armGroupLabels': ['Evinacumab']}]}, 'contactsLocationsModule': {'locations': [{'zip': '19803', 'city': 'Wilmington', 'state': 'Delaware', 'country': 'United States', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 39.74595, 'lon': -75.54659}}, {'zip': '33434', 'city': 'Boca Raton', 'state': 'Florida', 'country': 'United States', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 26.35869, 'lon': -80.0831}}, {'zip': '66190', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Regeneron Research Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '19106', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Regeneron Research Center', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'Regeneron Research Center', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Regeneron Research Center', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '1090', 'city': 'Vienna', 'country': 'Austria', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': '1105 AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': '11217', 'city': 'Taipei', 'country': 'Taiwan', 'facility': 'Regeneron Research Center', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'zip': '04209', 'city': 'Kyiv', 'country': 'Ukraine', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 50.45466, 'lon': 30.5238}}], 'overallOfficials': [{'name': 'Clinical Trial Management', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Regeneron Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR', 'ANALYTIC_CODE'], 'timeFrame': 'Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.', 'ipdSharing': 'YES', 'description': 'All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing', 'accessCriteria': 'Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency \\[EMA\\], Pharmaceuticals and Medical Devices Agency \\[PMDA\\], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Regeneron Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}