Ignite Creation Date:
2025-12-25 @ 12:39 AM
Ignite Modification Date:
2026-03-09 @ 2:22 AM
Study NCT ID:
NCT01617967
Status:
COMPLETED
Last Update Posted:
2024-04-19
First Post:
2012-06-07
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C000606954', 'term': 'patisiran'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@alnylam.com', 'phone': '1-877-256-9526', 'title': 'Chief Medical Officer', 'organization': 'Alnylam Pharmaceuticals, Inc.'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first dose of study drug up to Day 56', 'description': 'ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.', 'eventGroups': [{'id': 'EG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 1, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 2, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.', 'otherNumAtRisk': 7, 'deathsNumAtRisk': 7, 'otherNumAffected': 7, 'seriousNumAtRisk': 7, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG004', 'title': 'Patisiran 0.300 mg/kg Q3W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG005', 'title': 'Patisiran 0.300 mg/kg Q3W Alternative', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.', 'otherNumAtRisk': 9, 'deathsNumAtRisk': 9, 'otherNumAffected': 7, 'seriousNumAtRisk': 9, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Leukocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Neutrophilia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Tachyarrhythmia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Ventricular arrhythmia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Conjunctival haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Extravasation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Vessel puncture site haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Infusion related reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 3}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Herpes virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Lymphangitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Vulvovaginal mycotic infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Traumatic haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Hyperglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Temporomandibular joint syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Neuralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Presyncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Glycosuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Polyuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Renal impairment', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}], 'seriousEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 7, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 9, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (15.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '3', 'groupId': 'OG004'}, {'value': '9', 'groupId': 'OG005'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'Patisiran 0.300 mg/kg Q3W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).'}, {'id': 'OG005', 'title': 'Patisiran 0.300 mg/kg Q3W Alternative', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.'}], 'classes': [{'title': 'At Least 1 AE', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '3', 'groupId': 'OG004'}, {'value': '7', 'groupId': 'OG005'}]}]}, {'title': 'At Least 1 SAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '1', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}, {'value': '1', 'groupId': 'OG005'}]}]}, {'title': 'Study Drug Discontinuation For Any Reason', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}, {'value': '1', 'groupId': 'OG005'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Up to 56 days post first dose', 'description': 'The number of participants experiencing at least one adverse event (AE), at least one serious adverse event (SAE) and study drug discontinuation (due to any reason).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-to-treat (ITT) population included all participants, who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Percentage Change From Baseline in Serum Transthyretin (TTR) Protein', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '3', 'groupId': 'OG004'}, {'value': '8', 'groupId': 'OG005'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'Patisiran 0.300 mg/kg Q3W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).'}, {'id': 'OG005', 'title': 'Patisiran 0.300 mg/kg Q3W Alternative', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.'}], 'classes': [{'title': 'Day 21 or 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '3', 'groupId': 'OG004'}, {'value': '8', 'groupId': 'OG005'}]}], 'categories': [{'measurements': [{'value': '-8.9', 'spread': '9.91', 'groupId': 'OG000'}, {'value': '-21.7', 'spread': '1.69', 'groupId': 'OG001'}, {'value': '-51.6', 'spread': '22.84', 'groupId': 'OG002'}, {'value': '-73.9', 'spread': '8.41', 'groupId': 'OG003'}, {'value': '-78.0', 'spread': '7.55', 'groupId': 'OG004'}, {'value': '-80.7', 'spread': '10.14', 'groupId': 'OG005'}]}]}, {'title': 'Day 42 or 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}, {'value': '3', 'groupId': 'OG004'}, {'value': '8', 'groupId': 'OG005'}]}], 'categories': [{'measurements': [{'value': '-20.3', 'spread': '20.27', 'groupId': 'OG000'}, {'value': '-14.6', 'spread': '15.8', 'groupId': 'OG001'}, {'value': '-44.7', 'spread': '12.11', 'groupId': 'OG002'}, {'value': '-62.8', 'spread': '25.11', 'groupId': 'OG003'}, {'value': '-78.7', 'spread': '9.77', 'groupId': 'OG004'}, {'value': '-73.3', 'spread': '19.55', 'groupId': 'OG005'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Day 21/28 and Day 42/56 depending on dosing regimen (Q3W/Q4W)', 'description': 'Percentage change of TTR relative to pretreatment/baseline levels is reported. For arms with a dosing regimen of Q4W TTR protein samples were measured on Days 28 and 56. For the arms with a dosing regimen of Q3W TTR protein samples were measured on Days 21 and 42.', 'unitOfMeasure': 'Percentage', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants, who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '12', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'All Patisiran 0.300 mg/kg Q3W', 'description': 'All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.'}], 'classes': [{'title': 'Day 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '12', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '2738', 'spread': '3159', 'groupId': 'OG000'}, {'value': '9604', 'spread': '10588', 'groupId': 'OG001'}, {'value': '18998', 'spread': '5066', 'groupId': 'OG002'}, {'value': '53724', 'spread': '35814', 'groupId': 'OG003'}, {'value': '39741', 'spread': '33373', 'groupId': 'OG004'}]}]}, {'title': 'Day 21 or Day 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '2799', 'spread': '2645', 'groupId': 'OG000'}, {'value': '4884', 'spread': '4652', 'groupId': 'OG001'}, {'value': '27748', 'spread': '17081', 'groupId': 'OG002'}, {'value': '30013', 'spread': '15935', 'groupId': 'OG003'}, {'value': '25958', 'spread': '28460', 'groupId': 'OG004'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W)', 'description': 'Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.', 'unitOfMeasure': 'ng*h/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '12', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'All Patisiran 0.300 mg/kg Q3W', 'description': 'All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.'}], 'classes': [{'title': 'Day 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '12', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '145', 'spread': '23.4', 'groupId': 'OG000'}, {'value': '672', 'spread': '473', 'groupId': 'OG001'}, {'value': '2560', 'spread': '295', 'groupId': 'OG002'}, {'value': '6053', 'spread': '1326', 'groupId': 'OG003'}, {'value': '4539', 'spread': '1362', 'groupId': 'OG004'}]}]}, {'title': 'Day 21 or 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '106', 'spread': '36.6', 'groupId': 'OG000'}, {'value': '683', 'spread': '391', 'groupId': 'OG001'}, {'value': '3243', 'spread': '986', 'groupId': 'OG002'}, {'value': '3782', 'spread': '1259', 'groupId': 'OG003'}, {'value': '3314', 'spread': '1253', 'groupId': 'OG004'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W)', 'description': 'Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'All Patisiran 0.300 mg/kg Q3W', 'description': 'All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.'}], 'classes': [{'title': 'Day 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '59.5', 'spread': '59.3', 'groupId': 'OG000'}, {'value': '39.6', 'spread': '34.8', 'groupId': 'OG001'}, {'value': '52.2', 'spread': 'NA', 'comment': 'Standard deviation not calculated for 2 participants', 'groupId': 'OG002'}, {'value': '48', 'spread': '23.7', 'groupId': 'OG003'}, {'value': '59.3', 'spread': '17.2', 'groupId': 'OG004'}]}]}, {'title': 'Day 21 or 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}, {'value': '9', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '88.1', 'spread': 'NA', 'comment': 'Standard deviation not calculated for 2 participants', 'groupId': 'OG000'}, {'value': '39.4', 'spread': '34.8', 'groupId': 'OG001'}, {'value': '46.9', 'spread': '6.05', 'groupId': 'OG002'}, {'value': '50.2', 'spread': '32.7', 'groupId': 'OG003'}, {'value': '53.8', 'spread': '15.2', 'groupId': 'OG004'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.', 'unitOfMeasure': 'hour (h)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'All Patisiran 0.300 mg/kg Q3W', 'description': 'All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.'}], 'classes': [{'title': 'Day 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '0.0163', 'spread': '0.0248', 'groupId': 'OG000'}, {'value': '0.123', 'spread': '0.205', 'groupId': 'OG001'}, {'value': '0.00883', 'spread': 'NA', 'comment': 'Standard deviation not calculated for 2 participants', 'groupId': 'OG002'}, {'value': '0.00851', 'spread': '0.00606', 'groupId': 'OG003'}, {'value': '0.0130', 'spread': '0.0105', 'groupId': 'OG004'}]}]}, {'title': 'Day 21 or 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '6', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '0.0211', 'spread': '0.0325', 'groupId': 'OG000'}, {'value': '0.0844', 'spread': '0.134', 'groupId': 'OG001'}, {'value': '0.00658', 'spread': '0.00324', 'groupId': 'OG002'}, {'value': '0.0190', 'spread': '0.0233', 'groupId': 'OG003'}, {'value': '0.0222', 'spread': '0.0138', 'groupId': 'OG004'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.', 'unitOfMeasure': 'L/h/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'All Patisiran 0.300 mg/kg Q3W', 'description': 'All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.'}], 'classes': [{'title': 'Day 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '0.169', 'spread': '0.092', 'groupId': 'OG000'}, {'value': '0.279', 'spread': '0.0896', 'groupId': 'OG001'}, {'value': '0.428', 'spread': 'NA', 'comment': 'Standard deviation not calculated for 2 participants', 'groupId': 'OG002'}, {'value': '0.360', 'spread': '0.197', 'groupId': 'OG003'}, {'value': '0.588', 'spread': '0.305', 'groupId': 'OG004'}]}]}, {'title': 'Day 21 or 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '5', 'groupId': 'OG003'}, {'value': '9', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '0.264', 'spread': 'NA', 'comment': 'Standard deviation not calculated for 2 participants', 'groupId': 'OG000'}, {'value': '0.397', 'spread': 'NA', 'comment': 'Standard deviation not calculated for 2 participants', 'groupId': 'OG001'}, {'value': '0.305', 'spread': '0.163', 'groupId': 'OG002'}, {'value': '0.587', 'spread': '0.251', 'groupId': 'OG003'}, {'value': '0.881', 'spread': '0.539', 'groupId': 'OG004'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.', 'unitOfMeasure': 'L/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'OG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'OG004', 'title': 'All Patisiran 0.300 mg/kg Q3W', 'description': 'All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.'}], 'classes': [{'title': 'Day 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '7', 'groupId': 'OG003'}, {'value': '11', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '0.0153', 'spread': '0.0307', 'groupId': 'OG000'}, {'value': '0.0268', 'spread': '0.0465', 'groupId': 'OG001'}, {'value': '0.0129', 'spread': '0.0223', 'groupId': 'OG002'}, {'value': '0.0329', 'spread': '0.0500', 'groupId': 'OG003'}, {'value': '0.0334', 'spread': '0.0631', 'groupId': 'OG004'}]}]}, {'title': 'Day 21 or 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}, {'value': '5', 'groupId': 'OG003'}, {'value': '9', 'groupId': 'OG004'}]}], 'categories': [{'measurements': [{'value': '0.0320', 'spread': '0.0555', 'groupId': 'OG000'}, {'value': '0.253', 'spread': '0.439', 'groupId': 'OG001'}, {'value': '0.0410', 'spread': '0.0710', 'groupId': 'OG002'}, {'value': '0.0652', 'spread': '0.0893', 'groupId': 'OG003'}, {'value': '0.0563', 'spread': '0.0979', 'groupId': 'OG004'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose (within 1 h of planned dosing start) and post-infusion at 0-6 h (pooled) on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.', 'unitOfMeasure': 'mL/h/kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'FG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'FG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'FG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'FG004', 'title': 'Patisiran 0.300 mg/kg Q3W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).'}, {'id': 'FG005', 'title': 'Patisiran 0.300 mg/kg Q3W Alternative', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '3'}, {'groupId': 'FG003', 'numSubjects': '7'}, {'groupId': 'FG004', 'numSubjects': '3'}, {'groupId': 'FG005', 'numSubjects': '9'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '3'}, {'groupId': 'FG003', 'numSubjects': '6'}, {'groupId': 'FG004', 'numSubjects': '3'}, {'groupId': 'FG005', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '1'}, {'groupId': 'FG004', 'numSubjects': '0'}, {'groupId': 'FG005', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '1'}, {'groupId': 'FG004', 'numSubjects': '0'}, {'groupId': 'FG005', 'numSubjects': '0'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}, {'groupId': 'FG004', 'numSubjects': '0'}, {'groupId': 'FG005', 'numSubjects': '1'}]}, {'type': 'Dosing Suspended by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}, {'groupId': 'FG004', 'numSubjects': '0'}, {'groupId': 'FG005', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'A total of 29 subjects were enrolled'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '7', 'groupId': 'BG003'}, {'value': '3', 'groupId': 'BG004'}, {'value': '9', 'groupId': 'BG005'}, {'value': '29', 'groupId': 'BG006'}]}], 'groups': [{'id': 'BG000', 'title': 'Patisiran 0.010 mg/kg Q4W', 'description': 'Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).'}, {'id': 'BG001', 'title': 'Patisiran 0.050 mg/kg Q4W', 'description': 'Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'BG002', 'title': 'Patisiran 0.150 mg/kg Q4W', 'description': 'Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'BG003', 'title': 'Patisiran 0.300 mg/kg Q4W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.'}, {'id': 'BG004', 'title': 'Patisiran 0.300 mg/kg Q3W', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).'}, {'id': 'BG005', 'title': 'Patisiran 0.300 mg/kg Q3W Alternative', 'description': 'Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.'}, {'id': 'BG006', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '65.8', 'spread': '8.96', 'groupId': 'BG000'}, {'value': '55.7', 'spread': '24.83', 'groupId': 'BG001'}, {'value': '41.7', 'spread': '2.52', 'groupId': 'BG002'}, {'value': '58.7', 'spread': '16.07', 'groupId': 'BG003'}, {'value': '51.0', 'spread': '16.09', 'groupId': 'BG004'}, {'value': '54.7', 'spread': '16.34', 'groupId': 'BG005'}, {'value': '55.6', 'spread': '15.61', 'groupId': 'BG006'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}, {'value': '1', 'groupId': 'BG004'}, {'value': '2', 'groupId': 'BG005'}, {'value': '9', 'groupId': 'BG006'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}, {'value': '2', 'groupId': 'BG004'}, {'value': '7', 'groupId': 'BG005'}, {'value': '20', 'groupId': 'BG006'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '0', 'groupId': 'BG005'}, {'value': '0', 'groupId': 'BG006'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '0', 'groupId': 'BG005'}, {'value': '0', 'groupId': 'BG006'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '0', 'groupId': 'BG005'}, {'value': '0', 'groupId': 'BG006'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '0', 'groupId': 'BG005'}, {'value': '0', 'groupId': 'BG006'}]}, {'title': 'White', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '7', 'groupId': 'BG003'}, {'value': '3', 'groupId': 'BG004'}, {'value': '9', 'groupId': 'BG005'}, {'value': '29', 'groupId': 'BG006'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '0', 'groupId': 'BG005'}, {'value': '0', 'groupId': 'BG006'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '0', 'groupId': 'BG005'}, {'value': '0', 'groupId': 'BG006'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2013-01-16', 'size': 3080092, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2018-08-30T17:25', 'hasProtocol': True}, {'date': '2013-07-23', 'size': 1817116, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_000.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2018-08-30T16:11', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-05'}, 'expandedAccessInfo': {'nctId': 'NCT02939820', 'statusForNctId': 'APPROVED_FOR_MARKETING', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2018-10', 'dispFirstSubmitDate': '2017-06-02', 'completionDateStruct': {'date': '2014-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-04-17', 'studyFirstSubmitDate': '2012-06-07', 'dispFirstSubmitQcDate': '2017-07-13', 'resultsFirstSubmitDate': '2018-08-30', 'studyFirstSubmitQcDate': '2012-06-11', 'dispFirstPostDateStruct': {'date': '2017-07-17', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2024-04-19', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-10-23', 'studyFirstPostDateStruct': {'date': '2012-06-13', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-11-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2013-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation', 'timeFrame': 'Up to 56 days post first dose', 'description': 'The number of participants experiencing at least one adverse event (AE), at least one serious adverse event (SAE) and study drug discontinuation (due to any reason).'}], 'secondaryOutcomes': [{'measure': 'Percentage Change From Baseline in Serum Transthyretin (TTR) Protein', 'timeFrame': 'Baseline to Day 21/28 and Day 42/56 depending on dosing regimen (Q3W/Q4W)', 'description': 'Percentage change of TTR relative to pretreatment/baseline levels is reported. For arms with a dosing regimen of Q4W TTR protein samples were measured on Days 28 and 56. For the arms with a dosing regimen of Q3W TTR protein samples were measured on Days 21 and 42.'}, {'measure': 'Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last)', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W)', 'description': 'Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.'}, {'measure': 'Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax)', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W)', 'description': 'Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.'}, {'measure': 'Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta)', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.'}, {'measure': 'Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL)', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.'}, {'measure': 'Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss)', 'timeFrame': 'Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.'}, {'measure': 'Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR)', 'timeFrame': 'Predose (within 1 h of planned dosing start) and post-infusion at 0-6 h (pooled) on Day 0 and Day 21/28 depending on dosing frequency', 'description': 'Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True}, 'conditionsModule': {'keywords': ['RNAi therapeutic'], 'conditions': ['TTR-mediated Amyloidosis']}, 'referencesModule': {'references': [{'pmid': '26338094', 'type': 'DERIVED', 'citation': 'Suhr OB, Coelho T, Buades J, Pouget J, Conceicao I, Berk J, Schmidt H, Waddington-Cruz M, Campistol JM, Bettencourt BR, Vaishnaw A, Gollob J, Adams D. Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J Rare Dis. 2015 Sep 4;10:109. doi: 10.1186/s13023-015-0326-6.'}]}, 'descriptionModule': {'briefSummary': 'This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '85 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Body mass index must be between 17 kg/m\\^2 and ≤ 33 kg/m\\^2;\n* Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use appropriate contraception;\n* Males agree to use appropriate contraception;\n* Diagnosis of TTR amyloidosis;\n* Adequate blood counts, liver and renal function;\n* Willing to give written informed consent and are willing to comply with the study requirements.\n\nExclusion Criteria:\n\n* Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;\n* Received an investigational agent, other than tafamidis or diflunisal, within 30 days prior to first dose study drug administration;\n* Prior liver transplant;\n* Poor cardiac function;\n* Considered unfit for the study by the Principal Investigator;\n* Employee or family member of the sponsor or the clinical study site personnel.'}, 'identificationModule': {'nctId': 'NCT01617967', 'briefTitle': 'Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alnylam Pharmaceuticals'}, 'officialTitle': 'A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients With TTR Amyloidosis', 'orgStudyIdInfo': {'id': 'ALN-TTR02-002'}, 'secondaryIdInfos': [{'id': '2012-000467-24', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Patisiran (ALN-TTR02)', 'description': 'Two administrations of patisiran will be administered once every 4 weeks \\[Q4W\\]) in 4 sequential cohorts with escalating doses followed by optional cohorts with an alternative dosing regimen (once every 3 weeks \\[Q3W\\]), and an alternative premedication regimen.', 'interventionNames': ['Drug: Patisiran']}], 'interventions': [{'name': 'Patisiran', 'type': 'DRUG', 'otherNames': ['ALN-TTR02'], 'description': 'Participants received a single dose of patisiran as an intravenous (IV) infusion on Day 0 and Day 28 (Q4W). Optional cohorts received an alternative dosing regimen (once every 3 weeks \\[Q3W\\]: Day 0 and Day 21) and an alternative premedication regimen.', 'armGroupLabels': ['Patisiran (ALN-TTR02)']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'city': 'Rio de Janeiro', 'country': 'Brazil', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': -22.90642, 'lon': -43.18223}}, {'city': 'Le Kremlin-Bicêtre', 'country': 'France', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 48.81471, 'lon': 2.36073}}, {'city': 'Marseille', 'country': 'France', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'city': 'Münster', 'country': 'Germany', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.96236, 'lon': 7.62571}}, {'city': 'Lisbon', 'country': 'Portugal', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 38.72509, 'lon': -9.1498}}, {'city': 'Porto', 'country': 'Portugal', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.1485, 'lon': -8.61097}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'city': 'Palma de Mallorca', 'country': 'Spain', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.56939, 'lon': 2.65024}}, {'city': 'Umeå', 'country': 'Sweden', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 63.82842, 'lon': 20.25972}}], 'overallOfficials': [{'name': 'Jared Gollob, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Alnylam Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': 'Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU.\n\nData will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alnylam Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}