Ignite Creation Date:
2025-12-24 @ 4:33 PM
Ignite Modification Date:
2026-01-31 @ 1:26 AM
Study NCT ID:
NCT03625466
Status:
COMPLETED
Last Update Posted:
2022-11-02
First Post:
2018-08-07
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000599212', 'term': 'lumacaftor, ivacaftor drug combination'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medicalinfo@vrtx.com', 'phone': '617-341-6777', 'title': 'Medical Monitor', 'organization': 'Vertex Pharmaceuticals Incorporated'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From Day 1 up to Week 98 (Part 1: From Day 1 up to Week 48; Part 2: From Week 48 up to Week 98)', 'description': 'MedDRA 23.1 applied for Part 1 and MedDRA 24.0 applied for Part 2.', 'eventGroups': [{'id': 'EG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.', 'otherNumAtRisk': 16, 'deathsNumAtRisk': 16, 'otherNumAffected': 16, 'seriousNumAtRisk': 16, 'deathsNumAffected': 0, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing \\<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \\>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.', 'otherNumAtRisk': 35, 'deathsNumAtRisk': 35, 'otherNumAffected': 33, 'seriousNumAtRisk': 35, 'deathsNumAffected': 0, 'seriousNumAffected': 7}, {'id': 'EG002', 'title': 'Part 2: Overall LUM/IVA', 'description': 'Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \\<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \\>=6 years of age at week 48) in open-label period for 48 weeks.', 'otherNumAtRisk': 49, 'deathsNumAtRisk': 49, 'otherNumAffected': 41, 'seriousNumAtRisk': 49, 'deathsNumAffected': 0, 'seriousNumAffected': 12}], 'otherEvents': [{'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 7}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Post-tussive vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Bacterial disease carrier', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Enterobiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 15}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 11}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 22}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 19}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 9}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Traumatic haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Staphylococcus test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 10}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 11}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 8}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Otorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Astigmatism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Blepharospasm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Abnormal faeces', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Faeces pale', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Frequent bowel movements', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Peripheral swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Diarrhoea infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Febrile infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Otitis media acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pneumonia pseudomonal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Skin laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pseudomonas test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Lung infiltration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Nasal obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Nasal polyps', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Dry skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}], 'seriousEvents': [{'term': 'Coeliac disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Distal intestinal obstruction syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Bronchopulmonary aspergillosis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Chronic sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pseudomonas bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Sedation complication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Lung infiltration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Haematemesis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Intussusception', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 35, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 49, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1, 24.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '32', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.'}, {'id': 'OG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing \\<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \\>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.3', 'spread': '6.1', 'groupId': 'OG000'}, {'value': '-1.7', 'spread': '6.6', 'groupId': 'OG001'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.5', 'ciLowerLimit': '-5.5', 'ciUpperLimit': '2.6', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline at Week 48', 'description': 'MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = \\<50% of lobe involved and 2 = \\>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set (FAS) included all randomized participants who carried the intended CF transmembrane conductance regulator gene (CFTR) allele mutation and received at least 1 dose of study drug in Part 1. Here "overall number of participants analyzed" signifies participants who were evaluable for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.'}, {'id': 'OG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing \\<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \\>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.32', 'groupId': 'OG000', 'lowerLimit': '-0.20', 'upperLimit': '0.84'}, {'value': '-0.37', 'groupId': 'OG001', 'lowerLimit': '-0.85', 'upperLimit': '0.10'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline Through Week 48', 'description': 'LCI2.5 represents the number of lung turnovers required to reduce the end-tidal inert gas concentration to 1/40th of its starting value.', 'unitOfMeasure': 'lung clearance index', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS.'}, {'type': 'SECONDARY', 'title': 'Part 1: Absolute Change in Weight-for-age Z-score at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.'}, {'id': 'OG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing \\<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \\>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.07', 'groupId': 'OG000', 'lowerLimit': '-0.24', 'upperLimit': '0.11'}, {'value': '0.13', 'groupId': 'OG001', 'lowerLimit': '-0.01', 'upperLimit': '0.27'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline at Week 48', 'description': 'The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.', 'unitOfMeasure': 'z-score', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS.'}, {'type': 'SECONDARY', 'title': 'Part 1: Absolute Change in Stature-for-age Z-score at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.'}, {'id': 'OG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing \\<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \\>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.10', 'groupId': 'OG000', 'lowerLimit': '-0.04', 'upperLimit': '0.24'}, {'value': '0.09', 'groupId': 'OG001', 'lowerLimit': '-0.05', 'upperLimit': '0.22'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline at Week 48', 'description': 'The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.', 'unitOfMeasure': 'z-score', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS.'}, {'type': 'SECONDARY', 'title': 'Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.'}, {'id': 'OG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing \\<14 kg at screening received LUM 100 mg/IVA 125 mg FDC q12h in placebo-controlled period for 48 weeks. Participants weighing \\>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.24', 'groupId': 'OG000', 'lowerLimit': '-0.55', 'upperLimit': '0.07'}, {'value': '0.20', 'groupId': 'OG001', 'lowerLimit': '-0.02', 'upperLimit': '0.41'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline at Week 48', 'description': 'BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.', 'unitOfMeasure': 'z-score', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.'}, {'id': 'FG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing less than (\\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.'}, {'id': 'FG002', 'title': 'Part 2: Overall LUM/IVA', 'description': 'Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \\<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \\>=6 years of age at week 48) in open-label period for 48 weeks.'}], 'periods': [{'title': 'Placebo-controlled Period (48 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}, {'groupId': 'FG001', 'numSubjects': '35'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}, {'groupId': 'FG001', 'numSubjects': '33'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Withdrawal of Consent (not due to AE)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}, {'title': 'Open-label Period (48 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '49'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '48'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Withdrawal of Consent (not due to AE)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'This study was planned in 2 parts: Part 1 (Placebo-controlled Period) and Part 2 (Open-label Period). The primary and secondary efficacy analyses were planned for only Part 1.', 'preAssignmentDetails': 'This study was conducted in participants with cystic fibrosis (CF) aged 2 to 5 years.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.'}, {'id': 'BG001', 'title': 'Part 1: LUM/IVA', 'description': 'Participants weighing \\<14 kg at screening received LUM 100 mg/IVA 125 mg FDC every q12h in placebo-controlled period for 48 weeks. Participants weighing \\>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '4.2', 'spread': '1.0', 'groupId': 'BG000'}, {'value': '4.2', 'spread': '1.0', 'groupId': 'BG001'}, {'value': '4.2', 'spread': '1.0', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '33', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '51', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Magnetic Resonance Imaging (MRI) Global Chest Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}, {'value': '34', 'groupId': 'BG001'}, {'value': '49', 'groupId': 'BG002'}]}], 'categories': [{'measurements': [{'value': '21.4', 'spread': '9.3', 'groupId': 'BG000'}, {'value': '17.6', 'spread': '9.7', 'groupId': 'BG001'}, {'value': '18.8', 'spread': '9.6', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1)Bronchiectasis/wall thickening 2)Mucus plugging 3)Abscesses/sacculations 4)Consolidations 5)Special findings 6)Mosaic pattern 7)Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe :0=normal value, 1=\\<50 percent(%) of lobe involved and 2=\\>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Baseline population included all randomized participants who have received at least 1 dose of study drug. Here "number analyzed" signifies participants who were evaluable for this outcome measure at the specified time point.'}], 'populationDescription': 'Baseline population included all randomized participants who have received at least 1 dose of study drug.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-07-24', 'size': 830078, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2021-10-08T02:50', 'hasProtocol': True}, {'date': '2020-10-16', 'size': 5072937, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2021-10-08T02:52', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Double blinded in Part 1, and Open label in Part 2'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 51}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-08-10', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2022-10', 'completionDateStruct': {'date': '2021-10-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-10-06', 'studyFirstSubmitDate': '2018-08-07', 'resultsFirstSubmitDate': '2021-10-08', 'studyFirstSubmitQcDate': '2018-08-07', 'lastUpdatePostDateStruct': {'date': '2022-11-02', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-11-08', 'studyFirstPostDateStruct': {'date': '2018-08-10', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2021-12-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-10-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48', 'timeFrame': 'From Baseline at Week 48', 'description': 'MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = \\<50% of lobe involved and 2 = \\>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.'}], 'secondaryOutcomes': [{'measure': 'Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48', 'timeFrame': 'From Baseline Through Week 48', 'description': 'LCI2.5 represents the number of lung turnovers required to reduce the end-tidal inert gas concentration to 1/40th of its starting value.'}, {'measure': 'Part 1: Absolute Change in Weight-for-age Z-score at Week 48', 'timeFrame': 'From Baseline at Week 48', 'description': 'The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.'}, {'measure': 'Part 1: Absolute Change in Stature-for-age Z-score at Week 48', 'timeFrame': 'From Baseline at Week 48', 'description': 'The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.'}, {'measure': 'Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48', 'timeFrame': 'From Baseline at Week 48', 'description': 'BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '39173175', 'type': 'DERIVED', 'citation': 'Stahl M, Roehmel J, Eichinger M, Doellinger F, Naehrlich L, Kopp MV, Dittrich AM, Sommerburg O, Ray P, Maniktala A, Xu T, Conner S, Joshi A, Mascia M, Wielputz MO, Mall MA. Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial. Ann Am Thorac Soc. 2024 Nov;21(11):1550-1559. doi: 10.1513/AnnalsATS.202402-201OC.'}, {'pmid': '36943405', 'type': 'DERIVED', 'citation': 'Stahl M, Roehmel J, Eichinger M, Doellinger F, Naehrlich L, Kopp MV, Dittrich AM, Lee C, Sommerburg O, Tian S, Xu T, Wu P, Joshi A, Ray P, Duncan ME, Wielputz MO, Mall MA. Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for F508del-CFTR: A Phase 2 Placebo-controlled Clinical Trial. Ann Am Thorac Soc. 2023 Aug;20(8):1144-1155. doi: 10.1513/AnnalsATS.202208-684OC.'}]}, 'descriptionModule': {'briefSummary': 'This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '5 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Subjects with confirmed diagnosis of CF.\n* Homozygous for F508del (F/F).\n* Subjects who weigh ≥8 kg without shoes and wearing light clothing at the Screening Visit.\n\nKey Exclusion Criteria:\n\n* Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.\n* Solid organ or hematological transplantation.\n* History of any illness or comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study.\n\nOther protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT03625466', 'briefTitle': 'A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'An Exploratory Phase 2, 2-part, Randomized, Double-blind, Placebo-controlled Study With a Long-term, Open-label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del', 'orgStudyIdInfo': {'id': 'VX16-809-121'}, 'secondaryIdInfos': [{'id': '2017-003761-99', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Part 1: Placebo', 'description': 'Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Part 1: LUM/IVA', 'description': 'Participants weighing less than (\\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.', 'interventionNames': ['Drug: LUM/IVA']}, {'type': 'EXPERIMENTAL', 'label': 'Part 2: Overall LUM/IVA', 'description': 'Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \\<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \\>=6 years of age at week 48) in open-label period for 48 weeks.', 'interventionNames': ['Drug: LUM/IVA']}], 'interventions': [{'name': 'LUM/IVA', 'type': 'DRUG', 'otherNames': ['Lumacaftor/Ivacaftor', 'VX-809/VX-770'], 'description': 'FDC tablets or granules for oral administration.', 'armGroupLabels': ['Part 2: Overall LUM/IVA']}, {'name': 'LUM/IVA', 'type': 'DRUG', 'otherNames': ['Lumacaftor/Ivacaftor', 'VX-809/VX-770'], 'description': 'FDC granules for oral administration.', 'armGroupLabels': ['Part 1: LUM/IVA']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo matched to LUM/IVA for oral administration.', 'armGroupLabels': ['Part 1: Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Berlin', 'country': 'Germany', 'facility': 'Charite Paediatric Pulmonology Department', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Giessen', 'country': 'Germany', 'facility': 'Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin', 'geoPoint': {'lat': 50.58727, 'lon': 8.67554}}, {'city': 'Hanover', 'country': 'Germany', 'facility': 'Hannover Medical School', 'geoPoint': {'lat': 52.37052, 'lon': 9.73322}}, {'city': 'Heidelberg', 'country': 'Germany', 'facility': 'Heidelberg Cystic Fibrosis Center', 'geoPoint': {'lat': 49.40768, 'lon': 8.69079}}, {'city': 'Lübeck', 'country': 'Germany', 'facility': 'Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin', 'geoPoint': {'lat': 53.86893, 'lon': 10.68729}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}