Viewing Study NCT02344004

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Ignite Creation Date: 2025-12-24 @ 7:57 PM
Ignite Modification Date: 2026-01-04 @ 6:00 AM
Study NCT ID: NCT02344004
Status: COMPLETED
Last Update Posted: 2020-05-07
First Post: 2015-01-09
Is NOT Gene Therapy: True
Has Adverse Events: True
Brief Title: Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone
Sponsor:
Organization:
Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Australia', 'Austria', 'Canada', 'France', 'Germany', 'Israel', 'Italy', 'Japan', 'Netherlands', 'New Zealand', 'Poland', 'South Korea', 'Spain', 'Sweden', 'Taiwan', 'Thailand', 'United Kingdom', 'United States']}, 'conditionBrowseModule': {'meshes': [{'id': 'D009165', 'term': 'Mycobacterium Infections, Nontuberculous'}], 'ancestors': [{'id': 'D009164', 'term': 'Mycobacterium Infections'}, {'id': 'D000193', 'term': 'Actinomycetales Infections'}, {'id': 'D016908', 'term': 'Gram-Positive Bacterial Infections'}, {'id': 'D001424', 'term': 'Bacterial Infections'}, {'id': 'D001423', 'term': 'Bacterial Infections and Mycoses'}, {'id': 'D007239', 'term': 'Infections'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001239', 'term': 'Inhalation'}], 'ancestors': [{'id': 'D015656', 'term': 'Respiratory Mechanics'}, {'id': 'D012119', 'term': 'Respiration'}, {'id': 'D012143', 'term': 'Respiratory Physiological Phenomena'}, {'id': 'D002943', 'term': 'Circulatory and Respiratory Physiological Phenomena'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'kevin.mange@insmed.com', 'phone': '908-947-2651', 'title': 'Kevin Mange (Senior Vice President, Clinical Development & Medical Affairs, ALIS)', 'organization': 'Insmed Inc'}, 'certainAgreement': {'otherDetails': 'Per the signed Investigator Agreement in the study protocol and protocol amendments, the PI agreed "not to originate or use the name of Insmed Incorporated, or study drug code in any publicity, news release, or other public announcement, written or oral, whether to the public, press, or otherwise, relating to this protocol, to any amendment to the protocol, or to the performance of this protocol, without the prior written consent of Insmed Incorporated."', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Treatment-emergent adverse events (AEs) were assessed at Baseline (Day 1) and all subsequent study visits through Month 28 (end of study).', 'description': 'The analysis population consisted of the safety population, defined as all participants who received at least 1 dose of either LAI+ MDR (multi-drug regimen) or MDR alone.\n\nParticipants counted in all-cause mortality constitutes all deaths, regardless of whether or not there was an adverse event.', 'eventGroups': [{'id': 'EG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes', 'otherNumAtRisk': 223, 'deathsNumAtRisk': 223, 'otherNumAffected': 199, 'seriousNumAtRisk': 223, 'deathsNumAffected': 11, 'seriousNumAffected': 45}, {'id': 'EG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)', 'otherNumAtRisk': 112, 'deathsNumAtRisk': 112, 'otherNumAffected': 72, 'seriousNumAtRisk': 112, 'deathsNumAffected': 8, 'seriousNumAffected': 23}], 'otherEvents': [{'term': 'Dysphonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 140, 'numAffected': 104}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 111, 'numAffected': 85}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 20, 'numAffected': 17}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 59, 'numAffected': 47}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 10, 'numAffected': 10}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 48, 'numAffected': 37}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 17, 'numAffected': 14}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 30, 'numAffected': 24}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Chronic obstructive pulmonary disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 25, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 16, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Infective exacerbation of bronchiectasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 16, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 15, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 10, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 9, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 10, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 31, 'numAffected': 29}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 30, 'numAffected': 25}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 17, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 6, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 38, 'numAffected': 36}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 8, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 22, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Chest discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 13, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 15, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 16, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 25, 'numAffected': 22}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 16, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 19, 'numAffected': 18}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 22, 'numAffected': 18}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Hypoacusis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 17, 'numAffected': 15}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 8, 'numAffected': 8}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Sputum increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 15, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}], 'seriousEvents': [{'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 8, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Chronic obstructive pulmonary disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 12, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Infective exacerbation of bronchiectasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 8, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Cardiac failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Cardiogenic shock', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Microvascular coronary artery disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pneumatosis intestinalis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Adenocarcinoma gastric', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Colon adenoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Lung adenocarcinoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Breast cancer recurrent', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Major depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Drug hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Cachexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Spinal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Peripheral vascular disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Shock', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Performance status decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Hepatic function abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Radius fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Computerised tomogram thorax abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Urinary retention', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Interstitial lung disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pneumothorax', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pneumonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pulmonary cavitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Acute respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Alveolitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Maxillary sinus pseudocyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pulmonary embolism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Bronchial fistula', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Lung infiltration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pulmonary mass', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Respiratory acidosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 9, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Infective exacerbation of chronic obstructive airways disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Mycobacterium avium complex infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Lung infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 4, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Infectious pleural effusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Lung infection pseudomonal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Pneumonia pseudomonal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Scedosporium infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Superinfection bacterial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Mycetoma mycotic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Acute myocardial infarction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Hypercapnic coma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}, {'term': 'Exostosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 223, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 112, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA version 22.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg once daily (QD) in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'OG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'classes': [{'title': 'Converter', 'categories': [{'measurements': [{'value': '65', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}]}]}, {'title': 'Non-Converter', 'categories': [{'measurements': [{'value': '159', 'groupId': 'OG000'}, {'value': '102', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'groupDescription': 'The proportion of participants achieving culture conversion by Month 6 was analyzed using the Cochran-Mantel-Haenszel test, stratified by smoking status and prior multi-drug regimen. The treatment comparison was tested at two-sided significance level of 0.05. The null hypothesis assumed that culture conversion by Month 6 is independent of treatment, and the alternative hypothesis assumed that culture conversion by Month 6 is associated with treatment.', 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY', 'otherAnalysisDescription': 'The final analysis of the primary endpoint, the number of participants achieving culture conversion at by Month 6, was performed after the last participants completed Month 6 and his/her Month 6 sputum culture result was available.'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'by Month 6', 'description': 'Sputum specimens were collected at Screening (Visit 1), Baseline (Visit 2), and at Visits 3 (Month 1) through 8 (Month 6). A negative culture result reflected a negative culture result for all sputum samples collected at each visit. Participants met the primary endpoint of culture conversion by Month 6 if they had 3 consecutive monthly MAC-negative sputum cultures during the first 6 months of the study. A participant needed to achieve the first of 3 consecutive negative sputum cultures (that defined culture conversion) by Month 4 in order to meet the primary endpoint by Month 6. Each participant in the intent to treat (ITT) population (ie, all randomized participants) was classified as either a converter or non-converter by Month 6.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm.'}, {'type': 'PRIMARY', 'title': 'Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'OG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'classes': [{'title': 'Durable Conversion', 'categories': [{'measurements': [{'value': '36', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Non-Durable Conversion', 'categories': [{'measurements': [{'value': '188', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Cochran-Mantel-Haenszel', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'up to Month 19', 'description': 'Sputum specimens were collected at screening, baseline (Day 1), during treatment, and at Months 1, 3, 6, and 12 months off treatment. Culture conversion with durability was defined as achieving culture conversion by Month 6 and then having no more than 2 consecutive broth positive cultures and no Agar positive culture up to 3 months off treatment. Converters with missing broth or Agar sputum culture result after Month 6 up to 3 months off treatment were considered as not achieving culture conversion with durability except those participants who are unable to produce sputum despite reasonable efforts, as reported by source documentation. Participants who had relapse/recurrence, had "rescue" medication and/or died before reaching 3 months off treatment were considered as not achieving culture conversion with durability.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'OG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '223', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '424.2', 'spread': '1.12', 'groupId': 'OG000'}, {'value': '420.9', 'spread': '0.48', 'groupId': 'OG001'}]}]}, {'title': 'Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '223', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '420.6', 'spread': '4.73', 'groupId': 'OG000'}, {'value': '420.3', 'spread': '2.57', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline to Month 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '223', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-3.6', 'spread': '3.25', 'groupId': 'OG000'}, {'value': '-0.5', 'spread': '4.20', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.7804', 'groupIds': ['OG000', 'OG001'], 'groupDescription': 'This was analyzed using a mixed model repeated measures (MMRM) analysis of change from Baseline at Months 4\\&6. MMRM included treatment, month, treatment-by-month interaction, combination of smoking status \\& prior MDR (4 levels: Yes/Yes, Yes/No, No/Yes, and No/No) as fixed factors, baseline 6MWD as a covariate \\& baseline 6MWD-by-month interaction. An unstructured covariance matrix was used for the MMRM.', 'statisticalMethod': 'Mixed Model Repeated Measures (MMRM)', 'nonInferiorityType': 'SUPERIORITY', 'otherAnalysisDescription': '* Baseline is defined as the last non-missing value prior to first dose of study drug.\n* Statistics were obtained from an mixed-effects model repeated measures (MMRM) model with pattern-mixture modeling of missing values due to dropout, which included treatment, month, the treatment-by-month interaction, and the combination of smoking status and prior multidrug regimen as fixed factors, the baseline 6MWT distance as a covariate and baseline 6MWT distance-by-month interaction. MMRM included postbaseline data through Month 6.\n* For baseline, n is the number of participants with a baseline score and at least 1 postbaseline score. For Month 6, n is the number of participants with a baseline score and a postbaseline score at the summarized visit.'}], 'paramType': 'MEAN', 'timeFrame': 'at Month 6', 'description': "A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and at Month 6. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment. The analysis of the change from Baseline (Day 1) to Month 6 in the 6MWT distance was performed after the last participant completed Month 6 and his/her 6MWT distance data were available.", 'unitOfMeasure': 'meters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm.'}, {'type': 'SECONDARY', 'title': 'Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'OG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'classes': [{'title': '25th Percentile', 'categories': [{'measurements': [{'value': '2.13', 'groupId': 'OG000', 'lowerLimit': '1.83', 'upperLimit': '3.87'}, {'value': 'NA', 'comment': 'The 25th percentile time to conversion could not be estimated due to an insufficient proportion of patients achieving culture conversion by Month 6.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}, {'title': '50th Percentile', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'The 50th percentile time to conversion could not be estimated due to an insufficient proportion of patients achieving culture conversion by Month 6.', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'The 50th percentile time to conversion could not be estimated due to an insufficient proportion of patients achieving culture conversion by Month 6.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Cox Proportional Hazard', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '3.92', 'ciLowerLimit': '2.01', 'ciUpperLimit': '7.63', 'groupDescription': 'Kaplan Meier estimates for the distribution of time to culture conversion were constructed for treatment arms. The treatment comparison was made using the stratified log rank test for the ITT population. The estimated median time to culture conversion for each treatment arm was not estimable. The time to culture conversion was analyzed using Cox regression model to estimate hazards ratio.', 'statisticalMethod': 'Regression, Cox', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': 'by Month 6', 'description': 'The time to culture conversion was defined by the date of the first of at least 3 consecutive monthly culture specimens that were Mycobacterium avium complex (MAC)-negative.\n\nThe 25th percentile time to conversion is the estimated time taken for 25% of participants to convert.\n\nThe 50th percentile time to conversion is the estimated time taken for 50% of participants to convert.', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm.'}, {'type': 'SECONDARY', 'title': 'Number of Participants Achieving Sustained Culture Conversion at the End of Treatment (EOT) in the LAI + MDR Arm Compared to the MDR Arm Alone', 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'OG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'classes': [{'categories': [{'title': 'Sustained Conversion', 'measurements': [{'value': '41', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}, {'title': 'Non-Sustained Conversion', 'measurements': [{'value': '183', 'groupId': 'OG000'}, {'value': '109', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'up to Month 16', 'description': 'Sustained conversion was evaluated in participants who completed at least 12 months of treatment from the start of culture conversion. Sustained conversion was defined as conversion (3 consecutive negative monthly sputum samples) by Month 6 with no positive agar media culture or no more than 2 broth media cultures up to and including the time point. Participants who did not convert were considered non-sustained conversions.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm.'}, {'type': 'SECONDARY', 'title': 'Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'OG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'timeFrame': 'up to Month 16', 'description': "A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and up to EOT or Month 16. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment.", 'reportingStatus': 'POSTED', 'populationDescription': 'Due to the widely varying timepoints that makeup the EOT visit, this was not considered an appropriate analysis and was not performed.'}, {'type': 'SECONDARY', 'title': "Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score", 'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'OG000'}, {'value': '112', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'OG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'classes': [{'title': 'SGRQ Total Score at Baseline:', 'denoms': [{'units': 'Participants', 'counts': [{'value': '168', 'groupId': 'OG000'}, {'value': '104', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '36.555', 'spread': '21.3777', 'groupId': 'OG000'}, {'value': '38.409', 'spread': '21.5753', 'groupId': 'OG001'}]}]}, {'title': 'SGRQ Total Score at Month 6:', 'denoms': [{'units': 'Participants', 'counts': [{'value': '169', 'groupId': 'OG000'}, {'value': '106', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '38.715', 'spread': '22.3559', 'groupId': 'OG000'}, {'value': '37.368', 'spread': '23.6868', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline to Month 6:', 'denoms': [{'units': 'Participants', 'counts': [{'value': '168', 'groupId': 'OG000'}, {'value': '104', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2.009', 'spread': '13.4128', 'groupId': 'OG000'}, {'value': '-1.312', 'spread': '11.6216', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'at Month 6', 'description': "The SGRQ was completed before administration of study drug at Baseline (Day 1) and Months 3, 6, 8, and 12, and at the EOT visit and the 3 months off treatment visit. The SGRQ is a self-administered questionnaire that has been validated in participants with airways disease, specifically in participants with bronchiectasis. The SGRQ assesses health-related quality of life in participants with chronic pulmonary disease by evaluating 3 health domains: symptoms (distress caused by respiratory symptoms); activity (effects of disturbances on mobility and physical activity); and impacts (the effect of disease on factors such as employment, personal control of one's health, and need for medication). A composite total score is derived as the sum of domain scores for symptoms, activity, and impact (0 = best possible score and 100 = worst possible score). A within patient reduction from baseline in score of 4 units is generally recognized as a clinically meaningful improvement in quality of life.", 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population was the set of all randomized participants. Participants were classified according to their assigned treatment. There was a total of 336 participants in the ITT population, including 224 participants in the LAI + MDR arm and 112 participants in the MDR alone arm.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received Liposomal Amikacin for Inhalation (LAI) 590 mg once daily (QD) in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 American Thoracic Society/Infectious Diseases Society of America \\[ATS/IDSA\\] Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'FG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '224'}, {'groupId': 'FG001', 'numSubjects': '112'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '163'}, {'groupId': 'FG001', 'numSubjects': '98'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '61'}, {'groupId': 'FG001', 'numSubjects': '14'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '9'}, {'groupId': 'FG001', 'numSubjects': '7'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '22'}, {'groupId': 'FG001', 'numSubjects': '5'}]}, {'type': 'Rescue Medication', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Other Not Specified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '12'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'This study was conducted at 127 sites in 18 countries.', 'preAssignmentDetails': "Participant flow table reflects the disposition through end of the study (Month 28).\n\nThe deaths under Reasons for Discontinuation reflect the primary reason (according to investigator's judgment) that the participant discontinued and does not represent all deaths in the trial."}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines); LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes'}, {'id': 'BG001', 'title': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '64.6', 'spread': '9.59', 'groupId': 'BG000'}, {'value': '64.9', 'spread': '10.16', 'groupId': 'BG001'}, {'value': '64.7', 'spread': '9.77', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '165', 'groupId': 'BG000'}, {'value': '68', 'groupId': 'BG001'}, {'value': '233', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '59', 'groupId': 'BG000'}, {'value': '44', 'groupId': 'BG001'}, {'value': '103', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '211', 'groupId': 'BG000'}, {'value': '102', 'groupId': 'BG001'}, {'value': '313', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '58', 'groupId': 'BG000'}, {'value': '25', 'groupId': 'BG001'}, {'value': '83', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '158', 'groupId': 'BG000'}, {'value': '77', 'groupId': 'BG001'}, {'value': '235', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'If female, is participant of childbearing potential?', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '165', 'groupId': 'BG000'}, {'value': '68', 'groupId': 'BG001'}, {'value': '233', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Yes', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '19', 'groupId': 'BG002'}]}, {'title': 'No', 'measurements': [{'value': '152', 'groupId': 'BG000'}, {'value': '62', 'groupId': 'BG001'}, {'value': '214', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': '165/224 female participants in the LAI + Multi-drug regimen arm\n\n68/112 female participants in the Multi-drug regimen arm'}, {'title': 'If not childbearing potential, specify', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '152', 'groupId': 'BG000'}, {'value': '62', 'groupId': 'BG001'}, {'value': '214', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Postmenopausal', 'measurements': [{'value': '114', 'groupId': 'BG000'}, {'value': '50', 'groupId': 'BG001'}, {'value': '164', 'groupId': 'BG002'}]}, {'title': 'Surgically Sterile', 'measurements': [{'value': '38', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '50', 'groupId': 'BG002'}]}, {'title': 'Naturally Sterile', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': '152/224 female participants not of childbearing potential in the LAI + Multi-drug regimen arm\n\n62/112 female participants not of childbearing potential in the Multi-drug regimen arm'}, {'title': 'Region', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'title': 'North America', 'measurements': [{'value': '104', 'groupId': 'BG000'}, {'value': '55', 'groupId': 'BG001'}, {'value': '159', 'groupId': 'BG002'}]}, {'title': 'Asia (excluding Japan)', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}, {'title': 'Japan', 'measurements': [{'value': '34', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '48', 'groupId': 'BG002'}]}, {'title': 'Rest of World', 'measurements': [{'value': '72', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '109', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Multidrug regimen prior to enrollment', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'title': 'On treatment', 'measurements': [{'value': '201', 'groupId': 'BG000'}, {'value': '101', 'groupId': 'BG001'}, {'value': '302', 'groupId': 'BG002'}]}, {'title': 'Off treatment for at least 3 months', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '34', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Smoking status (includes e-cigarettes)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Current smoker', 'measurements': [{'value': '26', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '36', 'groupId': 'BG002'}]}, {'title': 'Not a current smoker', 'measurements': [{'value': '198', 'groupId': 'BG000'}, {'value': '102', 'groupId': 'BG001'}, {'value': '300', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Prior nebulized IV amikacin', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '224', 'groupId': 'BG000'}, {'value': '112', 'groupId': 'BG001'}, {'value': '336', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '24', 'groupId': 'BG000'}, {'value': '15', 'groupId': 'BG001'}, {'value': '39', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Participants who had received prior nebulized IV amikacin', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-02-22', 'size': 2714140, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_002.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2019-10-16T23:14', 'hasProtocol': True}, {'date': '2016-10-24', 'size': 7373768, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_003.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2019-10-16T23:27', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 336}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-05-27', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-02', 'completionDateStruct': {'date': '2019-04-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-04-22', 'studyFirstSubmitDate': '2015-01-09', 'resultsFirstSubmitDate': '2018-10-25', 'studyFirstSubmitQcDate': '2015-01-16', 'lastUpdatePostDateStruct': {'date': '2020-05-07', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-10-25', 'studyFirstPostDateStruct': {'date': '2015-01-22', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-11-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-07-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm', 'timeFrame': 'by Month 6', 'description': 'Sputum specimens were collected at Screening (Visit 1), Baseline (Visit 2), and at Visits 3 (Month 1) through 8 (Month 6). A negative culture result reflected a negative culture result for all sputum samples collected at each visit. Participants met the primary endpoint of culture conversion by Month 6 if they had 3 consecutive monthly MAC-negative sputum cultures during the first 6 months of the study. A participant needed to achieve the first of 3 consecutive negative sputum cultures (that defined culture conversion) by Month 4 in order to meet the primary endpoint by Month 6. Each participant in the intent to treat (ITT) population (ie, all randomized participants) was classified as either a converter or non-converter by Month 6.'}, {'measure': 'Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone', 'timeFrame': 'up to Month 19', 'description': 'Sputum specimens were collected at screening, baseline (Day 1), during treatment, and at Months 1, 3, 6, and 12 months off treatment. Culture conversion with durability was defined as achieving culture conversion by Month 6 and then having no more than 2 consecutive broth positive cultures and no Agar positive culture up to 3 months off treatment. Converters with missing broth or Agar sputum culture result after Month 6 up to 3 months off treatment were considered as not achieving culture conversion with durability except those participants who are unable to produce sputum despite reasonable efforts, as reported by source documentation. Participants who had relapse/recurrence, had "rescue" medication and/or died before reaching 3 months off treatment were considered as not achieving culture conversion with durability.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm', 'timeFrame': 'at Month 6', 'description': "A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and at Month 6. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment. The analysis of the change from Baseline (Day 1) to Month 6 in the 6MWT distance was performed after the last participant completed Month 6 and his/her 6MWT distance data were available."}, {'measure': 'Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm', 'timeFrame': 'by Month 6', 'description': 'The time to culture conversion was defined by the date of the first of at least 3 consecutive monthly culture specimens that were Mycobacterium avium complex (MAC)-negative.\n\nThe 25th percentile time to conversion is the estimated time taken for 25% of participants to convert.\n\nThe 50th percentile time to conversion is the estimated time taken for 50% of participants to convert.'}, {'measure': 'Number of Participants Achieving Sustained Culture Conversion at the End of Treatment (EOT) in the LAI + MDR Arm Compared to the MDR Arm Alone', 'timeFrame': 'up to Month 16', 'description': 'Sustained conversion was evaluated in participants who completed at least 12 months of treatment from the start of culture conversion. Sustained conversion was defined as conversion (3 consecutive negative monthly sputum samples) by Month 6 with no positive agar media culture or no more than 2 broth media cultures up to and including the time point. Participants who did not convert were considered non-sustained conversions.'}, {'measure': 'Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone', 'timeFrame': 'up to Month 16', 'description': "A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and up to EOT or Month 16. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment."}, {'measure': "Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score", 'timeFrame': 'at Month 6', 'description': "The SGRQ was completed before administration of study drug at Baseline (Day 1) and Months 3, 6, 8, and 12, and at the EOT visit and the 3 months off treatment visit. The SGRQ is a self-administered questionnaire that has been validated in participants with airways disease, specifically in participants with bronchiectasis. The SGRQ assesses health-related quality of life in participants with chronic pulmonary disease by evaluating 3 health domains: symptoms (distress caused by respiratory symptoms); activity (effects of disturbances on mobility and physical activity); and impacts (the effect of disease on factors such as employment, personal control of one's health, and need for medication). A composite total score is derived as the sum of domain scores for symptoms, activity, and impact (0 = best possible score and 100 = worst possible score). A within patient reduction from baseline in score of 4 units is generally recognized as a clinically meaningful improvement in quality of life."}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['ALIS', 'ARIKAYCE'], 'conditions': ['Mycobacterium Infections, Nontuberculous']}, 'referencesModule': {'references': [{'pmid': '41186908', 'type': 'DERIVED', 'citation': 'Yamazaki Y, Nakagawa T, Jumadilova Z, Yuen D, Villa R, Hasegawa N. Management and Resolution of Hypersensitivity Pneumonitis-Related Events in Japanese Patients Treated with Amikacin Liposome Inhalation Suspension in the CONVERT and INS-312 Clinical Trials. Infect Dis Ther. 2025 Nov 4. doi: 10.1007/s40121-025-01247-7. Online ahead of print.'}, {'pmid': '38277865', 'type': 'DERIVED', 'citation': 'Morimoto K, Nonaka M, Yamazaki Y, Nakagawa T, Takasaki J, Tsuyuguchi K, Kitada S, Jumadilova Z, Yuen DW, Ciesielska M, Hasegawa N. Amikacin liposome inhalation suspension for Mycobacterium avium complex pulmonary disease: A subgroup analysis of Japanese patients in the randomized, phase 3, CONVERT study. Respir Investig. 2024 Mar;62(2):284-290. doi: 10.1016/j.resinv.2023.12.012. Epub 2024 Jan 25.'}, {'pmid': '33887244', 'type': 'DERIVED', 'citation': 'Griffith DE, Thomson R, Flume PA, Aksamit TR, Field SK, Addrizzo-Harris DJ, Morimoto K, Hoefsloot W, Mange KC, Yuen DW, Ciesielska M, Wallace RJ Jr, van Ingen J, Brown-Elliott BA, Coulter C, Winthrop KL; CONVERT Study Group. Amikacin Liposome Inhalation Suspension for Refractory Mycobacterium avium Complex Lung Disease: Sustainability and Durability of Culture Conversion and Safety of Long-term Exposure. Chest. 2021 Sep;160(3):831-842. doi: 10.1016/j.chest.2021.03.070. Epub 2021 Apr 19.'}, {'pmid': '33595792', 'type': 'DERIVED', 'citation': 'Rubino CM, Onufrak NJ, van Ingen J, Griffith DE, Bhavnani SM, Yuen DW, Mange KC, Winthrop KL. Population Pharmacokinetic Evaluation of Amikacin Liposome Inhalation Suspension in Patients with Treatment-Refractory Nontuberculous Mycobacterial Lung Disease. Eur J Drug Metab Pharmacokinet. 2021 Mar;46(2):277-287. doi: 10.1007/s13318-020-00669-7. Epub 2021 Feb 17.'}, {'pmid': '30216086', 'type': 'DERIVED', 'citation': "Griffith DE, Eagle G, Thomson R, Aksamit TR, Hasegawa N, Morimoto K, Addrizzo-Harris DJ, O'Donnell AE, Marras TK, Flume PA, Loebinger MR, Morgan L, Codecasa LR, Hill AT, Ruoss SJ, Yim JJ, Ringshausen FC, Field SK, Philley JV, Wallace RJ Jr, van Ingen J, Coulter C, Nezamis J, Winthrop KL; CONVERT Study Group. Amikacin Liposome Inhalation Suspension for Treatment-Refractory Lung Disease Caused by Mycobacterium avium Complex (CONVERT). A Prospective, Open-Label, Randomized Study. Am J Respir Crit Care Med. 2018 Dec 15;198(12):1559-1569. doi: 10.1164/rccm.201807-1318OC."}]}, 'descriptionModule': {'briefSummary': 'A study to evaluate the effectiveness of Liposomal Amikacin for Inhalation (LAI) 590 mg administered once daily (QD) when added to multi-drug regimen (MDR) in participants with Nontuberculous Mycobacterial (NTM) lung infection caused by Mycobacterium Avium Complex (MAC) that were refractory to treatment.\n\nParticipants were randomized 2:1 to LAI 590 mg administered QD + MDR or MDR alone.', 'detailedDescription': 'This is a randomized, open-label, multicenter study of LAI in adult participants with NTM lung infections caused by MAC that were refractory to treatment. Participants received either LAI 590 mg administered QD by inhalation plus a multidrug regimen, hereafter referred to as LAI + MDR or a multidrug regimen alone, hereafter referred to as MDR alone for a minimum of 8 months. Participants who demonstrated culture conversion by Month 6 went on to complete a treatment course of 12 months, starting from the first of 3 negative cultures that defined culture conversion.\n\nSputum culture results were made available to the site after the Month 6 sputum result was known, in time for the Month 8 visit. Prior to the Month 8 visit, the culture results from Baseline to Month 6 inclusively were blinded to the site and Sponsor. The results were blinded to reduce the potential for bias in an open-label study. At Month 8 (-28 to +7 days), after all sputum culture results were made available to the site only, up to and including Month 6, participants were assessed as converters or non-converters.\n\nA converter was defined as a participant who had 3 consecutive monthly MAC-negative sputum cultures at any time within the first 6 months of the study.\n\n"Relapse or recurrence" was defined as having MAC-positive sputum cultures in liquid broth media (agar negative) for 3 or more consecutive months, or having at least 1 MAC-positive sputum culture on solid media (agar positive) after achieving culture conversion.\n\nA non-converter was defined as a participant who did not have 3 consecutive monthly MAC-negative sputum cultures at any time within the first 6 months of the study.\n\nAll converters remained in the study. Converters who, after culture conversion, subsequently had MAC-positive sputum cultures in liquid broth media (agar negative) for 1 or 2 consecutive months only by Month 6 also remained in the study. Participants who remained in the study continued their randomized treatment regimen until they completed a total of 12 months of treatment (EOT), starting from the first of 3 negative cultures that defined culture conversion. These participants returned after the EOT visit for 28 days, 3, 6, and 12 months off-treatment follow-up visits. The 12 months off-treatment follow-up visit was the end of study (EOS) visit. No NTM treatment was administered during the off-treatment phase.\n\nAt Month 8, all non-converters as assessed at the Month 6 visit were discontinued from Study INS-212. Participants who experienced a relapse or recurrence by Month 6 also discontinued from Study INS-212 at their Month 8 visit. These participants were potentially eligible to enter a separate open-label study of LAI (Study INS-312), provided all entry criteria were met for that study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Be continually positive for MAC on sputum culture while adhering to a multi-drug treatment regimen for a minimum duration of 6 months which is either ongoing or was completed no more than 12 months before screening\n2. Be diagnosed with MAC NTM lung infection with evidence of nodular bronchiectasis and/or fibrocavitary disease by chest CT\n3. Be willing to adhere to multi-drug treatment regimen during the course of the study\n\nExclusion Criteria:\n\n1. Patients with cystic fibrosis\n2. Positive pregnancy test or lactation at screening. All women of child bearing potential will be tested. Women not of childbearing potential are defined as postmenopausal (i.e., amenorrheic for at least 1 year), or surgically or naturally sterile.\n3. Active pulmonary tuberculosis requiring treatment at screening\n4. History of lung transplantation\n5. Prior exposure to LAI (including clinical study).'}, 'identificationModule': {'nctId': 'NCT02344004', 'acronym': 'CONVERT', 'briefTitle': 'Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone', 'organization': {'class': 'INDUSTRY', 'fullName': 'Insmed Incorporated'}, 'officialTitle': 'A Randomized, Open-label, Multicenter Study of Liposomal Amikacin for Inhalation (LAI) in Adult Patients With Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC) That Are Refractory to Treatment', 'orgStudyIdInfo': {'id': 'INS-212'}, 'secondaryIdInfos': [{'id': '2014-005010-31', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'NO_INTERVENTION', 'label': 'Multi-drug Regimen', 'description': 'Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 American Thoracic Society/Infectious Diseases Society of America \\[ATS/IDSA\\] Guidelines)'}, {'type': 'EXPERIMENTAL', 'label': 'LAI + Multi-drug Regimen', 'description': 'Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)\n\nLAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes', 'interventionNames': ['Drug: LAI (Liposomal Amikacin for Inhalation) 590 mg']}], 'interventions': [{'name': 'LAI (Liposomal Amikacin for Inhalation) 590 mg', 'type': 'DRUG', 'otherNames': ['ALIS (Amikacin Liposome Inhalation Suspension)', 'ARIKAYCE®'], 'description': 'LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes.', 'armGroupLabels': ['LAI + Multi-drug Regimen']}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Gina Eagle, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Insmed Incorporated'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Insmed Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}