Ignite Creation Date:
2025-12-25 @ 12:53 AM
Ignite Modification Date:
2026-02-24 @ 10:55 AM
Study NCT ID:
NCT02725567
Status:
COMPLETED
Last Update Posted:
2023-09-07
First Post:
2016-03-14
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
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': 'C545203', 'term': 'ivacaftor'}]}}, '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 Through Safety Follow-up Period (up to Day 70 for Part A and up to Week 38 for Part B + A/B)', 'eventGroups': [{'id': 'EG000', 'title': 'Part A: 3 to < 24 Months', 'description': 'Participants weighing 5 to \\<7 kg received 25 mg IVA, weighing 7 to \\<14 kg received 50 mg IVA, and participants weighing 14 to \\< 25 kg received 75 mg IVA q12h on days 1 through day 3 and 1 morning dose on day 4.', 'otherNumAtRisk': 19, 'deathsNumAtRisk': 19, 'otherNumAffected': 10, 'seriousNumAtRisk': 19, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Part B + A/B: 1 to <24 Months', 'description': 'Participants 4 to \\<6 months of age and ≥5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h for 24 weeks on Part B.\n\nFor Part A/B, participants 1 to \\<4 months weighing 3 kg to \\<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.', 'otherNumAtRisk': 43, 'deathsNumAtRisk': 43, 'otherNumAffected': 34, 'seriousNumAtRisk': 43, 'deathsNumAffected': 0, 'seriousNumAffected': 6}], 'otherEvents': [{'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Infantile spitting up', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Teething', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 12}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Hand-foot-and-mouth disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Head injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 8}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 7}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Blood pressure increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Pseudomonas test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Sleep disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 24}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 6}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 12}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Miliaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}], 'seriousEvents': [{'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Distal intestinal obstruction syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Bronchiolitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Eczema Coxsackium', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Eczema herpeticum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 43, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: 3 to < 24 Months', 'description': 'Participants weighing 5 to \\<7 kg received 25 mg IVA, weighing 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h on Days 1 through Day 3 and 1 morning dose on Day 4.'}], 'classes': [{'title': 'Participants with TEAEs', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'Participants with Serious TEAEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 1 through Day 70', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety set included all participants who received at least 1 dose of study drug in Part A. This outcome measure was planned only for Part A: 3 to \\<24 months arm.'}, {'type': 'PRIMARY', 'title': 'Part A: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: 3 to < 24 Months', 'description': 'Participants weighing 5 to \\<7 kg received 25 mg IVA, weighing 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h on Days 1 through Day 3 and 1 morning dose on Day 4.'}], 'classes': [{'title': 'Pre dose: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '654', 'spread': '531', 'groupId': 'OG000'}]}]}, {'title': 'Pre dose: M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1880', 'spread': '1160', 'groupId': 'OG000'}]}]}, {'title': 'Pre dose: M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2680', 'spread': '1990', 'groupId': 'OG000'}]}]}, {'title': '2-4 hrs post dose: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '956', 'spread': '497', 'groupId': 'OG000'}]}]}, {'title': '2-4 hrs post dose: M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1990', 'spread': '1160', 'groupId': 'OG000'}]}]}, {'title': '2-4 hrs post dose: M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2460', 'spread': '2110', 'groupId': 'OG000'}]}]}, {'title': '6-8 hrs post dose: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1040', 'spread': '623', 'groupId': 'OG000'}]}]}, {'title': '6-8 hrs post dose: M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2440', 'spread': '1260', 'groupId': 'OG000'}]}]}, {'title': '6-8 hrs post dose: M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2880', 'spread': '2180', 'groupId': 'OG000'}]}]}, {'title': '24-60 hrs post dose: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '129', 'spread': '68.8', 'groupId': 'OG000'}]}]}, {'title': '24-60 hrs post dose: M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '508', 'spread': '228', 'groupId': 'OG000'}]}]}, {'title': '24-60 hrs post dose: M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1100', 'spread': '982', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-dose, 2-4 hours, 6-8 hours, 24-60 hours post-dose', 'unitOfMeasure': 'nanogram per milliliter (ng/ml)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug in Part A. Here the "Number Analyzed" signifies those participants who were evaluable for the specified time point.'}, {'type': 'PRIMARY', 'title': 'Part B +A/B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B + A/B: 1 to <24 Months', 'description': 'Participants 4 to \\<6 months of age and ≥5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h for 24 weeks on Part B.\n\nFor Part A/B, participants 1 to \\<4 months weighing 3 kg to \\<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.'}], 'classes': [{'title': 'Participants with TEAEs', 'categories': [{'measurements': [{'value': '38', 'groupId': 'OG000'}]}]}, {'title': 'Participants with Serious (TEAEs)', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Day 1 through Week 38', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Set included all participants who received at least 1 dose of study drug. The safety and tolerability analyses were assessed for the overall treatment arm. Therefore, the analysis is reported in a overall Part B+ A/B: 1 to \\<24 months arm.'}, {'type': 'PRIMARY', 'title': 'Part A/B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A/B: 1 to <4 Months', 'description': 'Participants 1 to \\<4 months weighing 3 kg to \\<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.'}], 'classes': [{'title': 'Day 4 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '348', 'spread': '151', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '867', 'spread': '412', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1570', 'spread': '570', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (2-4 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '381', 'spread': '135', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (2-4 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '851', 'spread': '333', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (2-4 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1370', 'spread': '450', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (6-8 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '501', 'spread': '196', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (6-8 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1190', 'spread': '420', 'groupId': 'OG000'}]}]}, {'title': 'Day 4 (6-8 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1670', 'spread': '551', 'groupId': 'OG000'}]}]}, {'title': 'Day 15 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '191', 'spread': '134', 'groupId': 'OG000'}]}]}, {'title': 'Day 15 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '614', 'spread': '378', 'groupId': 'OG000'}]}]}, {'title': 'Day 15 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1510', 'spread': '1110', 'groupId': 'OG000'}]}]}, {'title': 'Week 4 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '316', 'spread': '130', 'groupId': 'OG000'}]}]}, {'title': 'Week 4 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1170', 'spread': '577', 'groupId': 'OG000'}]}]}, {'title': 'Week 4 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3370', 'spread': '2330', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '449', 'spread': '352', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1030', 'spread': '476', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2380', 'spread': '1500', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (2-4 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '523', 'spread': '262', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (2-4 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1360', 'spread': '982', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (2-4 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2100', 'spread': '1540', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (6-8 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '438', 'spread': '79.8', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (6-8 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1420', 'spread': '606', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (6-8 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2320', 'spread': '1330', 'groupId': 'OG000'}]}]}, {'title': 'Week 12 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '213', 'spread': '173', 'groupId': 'OG000'}]}]}, {'title': 'Week 12 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '906', 'spread': '660', 'groupId': 'OG000'}]}]}, {'title': 'Week 12 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2500', 'spread': '1760', 'groupId': 'OG000'}]}]}, {'title': 'Week 18 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '226', 'spread': '153', 'groupId': 'OG000'}]}]}, {'title': 'Week 18 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '815', 'spread': '470', 'groupId': 'OG000'}]}]}, {'title': 'Week 18 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2060', 'spread': '1190', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '276', 'spread': '137', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1120', 'spread': '496', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2380', 'spread': '799', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); Day 15 (pre-dose); Week 4 (pre-dose); Week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 12 (pre-dose); Week 18 (pre-dose) and Week 24 (pre-dose)', 'unitOfMeasure': 'ng/ml', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set included participants who received at least 1 dose of study drug in Part A/B. Here the "Number Analyzed" signifies those participants who were evaluable for the specified time point.'}, {'type': 'SECONDARY', 'title': 'Part B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: 4 to <24 Months', 'description': 'Participants 4 to \\<6 months of age and ≥5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h for 24 weeks on Part B.'}], 'classes': [{'title': 'Week 2 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '457', 'spread': '441', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1340', 'spread': '934', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1980', 'spread': '1790', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (2-4 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '812', 'spread': '726', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (2-4 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1560', 'spread': '1190', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (2-4 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1770', 'spread': '1970', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (6-8 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '969', 'spread': '705', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (6-8 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2210', 'spread': '1360', 'groupId': 'OG000'}]}]}, {'title': 'Week 2 (6-8 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2140', 'spread': '1880', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '404', 'spread': '376', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1220', 'spread': '782', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1720', 'spread': '1110', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (1 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '466', 'spread': '384', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (1 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1100', 'spread': '670', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (1 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1500', 'spread': '881', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (4 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '996', 'spread': '520', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (4 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2130', 'spread': '950', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 (4 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '33', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1750', 'spread': '1010', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (pre dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '301', 'spread': '204', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (pre dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1050', 'spread': '492', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (pre dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1600', 'spread': '783', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (2-4 hrs post dose): IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '794', 'spread': '480', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (2-4 hrs post dose): M1-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1540', 'spread': '783', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (2-4 hrs post dose): M6-IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1320', 'spread': '592', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 2 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 8 (pre-dose,1 hour, 4-hour post-dose); Week 24 (pre-dose, 2-4 hours post dose)', 'unitOfMeasure': 'ng/ml', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set included participants who received at least 1 dose of study drug in Part B. Here the "Number Analyzed" signifies those participants who were evaluable for the specified time point.'}, {'type': 'SECONDARY', 'title': 'Part B + A/B: Absolute Change From Baseline in Sweat Chloride', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B + A/B: 1 to < 24 Months', 'description': 'Participants 4 to \\<6 months of age and ≥5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h for 24 weeks on Part B.\n\nFor Part A/B, participants 1 to \\<4 months weighing 3 kg to \\<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.'}], 'classes': [{'categories': [{'measurements': [{'value': '-62.0', 'spread': '22.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline at Week 24', 'description': 'Sweat samples were collected using an approved collection device.', 'unitOfMeasure': 'millimole per liter (mmol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Full Analysis Set (FAS) included all enrolled participants who were exposed to any amount of study drug in Part B + A/B. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Part A: 3 to < 24 Months', 'description': 'Participants weighing 5 to less than (\\<) 7 kilogram (kg) received 25 milligram (mg) IVA (ivacaftor), 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA administered every 12 hours (q12h) on Days 1 through 3 and 1 morning dose on Day 4.'}, {'id': 'FG001', 'title': 'Part B + A/B: 1 to < 24 Months', 'description': 'Participants 4 to \\<6 months of age and weighing greater than or equal to (≥) 5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h for 24 weeks on Part B.\n\nFor Part A/B, participants 1 to \\<4 months weighing 3 kg to \\<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.'}], 'periods': [{'title': 'Part A (Up to 5 Days)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '19'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '19'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'Part B + A/B ( Up to 24 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'comment': 'Out of the 19 participants in the reporting arm Part A, 5 also participated in the reporting arm "Part B + Part A/B.', 'groupId': 'FG001', 'numSubjects': '43'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '40'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '3'}]}], 'dropWithdraws': [{'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal of Consent (not due to adverse event)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'This study was conducted in participants with cystic fibrosis (CF) who were less than (\\<) 24 months of age at Day 1 and have an ivacaftor-responsive CF transmembrane conductance regulator (CFTR) mutation.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ivacaftor (IVA)', 'description': 'Part A: Participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA administered q12h on Days 1 through 3 and 1 morning dose on Day 4.\n\nParticipants 4 to \\<6 months of age and ≥5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h for 24 weeks on Part B.\n\nFor Part A/B, participants 1 to \\<4 months weighing 3 kg to \\<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'title': 'Part A', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'BG000'}]}], 'categories': [{'title': '12 to <24 months', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': '6 to <12 months', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': '4 to <6 months', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': '1 to <4 months', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}, {'title': 'Part B+ A/B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'BG000'}]}], 'categories': [{'title': '12 to <24 months', 'measurements': [{'value': '19', 'groupId': 'BG000'}]}, {'title': '6 to <12 months', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': '4 to <6 months', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': '1 to <4 months', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.'}, {'title': 'Sex: Female, Male', 'classes': [{'title': 'Part A', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}]}, {'title': 'Part B + A/B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '22', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '21', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Part A', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '19', 'groupId': 'BG000'}]}, {'title': 'Not collected per local regulations', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Part B + A/B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '42', 'groupId': 'BG000'}]}, {'title': 'Not collected per local regulations', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Part A', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'BG000'}]}], 'categories': [{'title': 'White', 'measurements': [{'value': '19', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not collected per local Regulations', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Part B + A/B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'BG000'}]}], 'categories': [{'title': 'White', 'measurements': [{'value': '43', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not collected per local Regulations', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.'}], 'populationDescription': 'All participants who received at least one dose of the study drug during the treatment period were included in the baseline analysis.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-04-01', 'size': 3191090, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-06-19T04:38', 'hasProtocol': True}, {'date': '2022-12-07', 'size': 920665, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-06-19T04:39', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 57}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-06-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-08', 'completionDateStruct': {'date': '2022-06-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-08-14', 'studyFirstSubmitDate': '2016-03-14', 'resultsFirstSubmitDate': '2023-06-27', 'studyFirstSubmitQcDate': '2016-03-31', 'lastUpdatePostDateStruct': {'date': '2023-09-07', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-08-14', 'studyFirstPostDateStruct': {'date': '2016-04-01', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2023-09-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-06-28', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)', 'timeFrame': 'Day 1 through Day 70'}, {'measure': 'Part A: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)', 'timeFrame': 'Pre-dose, 2-4 hours, 6-8 hours, 24-60 hours post-dose'}, {'measure': 'Part B +A/B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)', 'timeFrame': 'Day 1 through Week 38'}, {'measure': 'Part A/B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)', 'timeFrame': 'Day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); Day 15 (pre-dose); Week 4 (pre-dose); Week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 12 (pre-dose); Week 18 (pre-dose) and Week 24 (pre-dose)'}], 'secondaryOutcomes': [{'measure': 'Part B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)', 'timeFrame': 'Week 2 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 8 (pre-dose,1 hour, 4-hour post-dose); Week 24 (pre-dose, 2-4 hours post dose)'}, {'measure': 'Part B + A/B: Absolute Change From Baseline in Sweat Chloride', 'timeFrame': 'From Baseline at Week 24', 'description': 'Sweat samples were collected using an approved collection device.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '33023304', 'type': 'DERIVED', 'citation': 'Davies JC, Wainwright CE, Sawicki GS, Higgins MN, Campbell D, Harris C, Panorchan P, Haseltine E, Tian S, Rosenfeld M. Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial. Am J Respir Crit Care Med. 2021 Mar 1;203(5):585-593. doi: 10.1164/rccm.202008-3177OC.'}, {'pmid': '29886024', 'type': 'DERIVED', 'citation': 'Rosenfeld M, Wainwright CE, Higgins M, Wang LT, McKee C, Campbell D, Tian S, Schneider J, Cunningham S, Davies JC; ARRIVAL study group. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med. 2018 Jul;6(7):545-553. doi: 10.1016/S2213-2600(18)30202-9. Epub 2018 Jun 7.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in participants with cystic fibrosis (CF) who are \\<24 months of age at treatment initiation and have an ivacaftor-responsive CF transmembrane conductance regulator (CFTR) gene mutation.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '24 Months', 'minimumAge': '1 Month', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed diagnosis of CF by sweat chloride value or CF mutation criteria.\n* Have 1 of the following 10 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H (eligible in regions where ivacaftor is approved for use). Part A/B group may also have other ivacaftor-responsive mutations.\n* Hematology, serum chemistry, and vital signs results at screening with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator.\n\nExclusion Criteria:\n\n* History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant\n* Colonization with organisms associated with a more rapid decline in pulmonary status at screening (Only for Parts A and B)\n* History of abnormal liver function or abnormal liver function at screening\n* History of solid organ or hematological transplantation\n* Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1\n* Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives before screening\n* Hemoglobin (Hgb) \\<9.5 g/dL at screening\n* Chronic kidney disease of Stage 3 or above\n* Presence of a non-congenital or progressive lens opacity or cataract at Screening\n\nOther protocol defined Inclusion/Exclusion Criteria may apply.'}, 'identificationModule': {'nctId': 'NCT02725567', 'briefTitle': 'A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation', 'orgStudyIdInfo': {'id': 'VX15-770-124'}, 'secondaryIdInfos': [{'id': '2015-001997-16', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part A: 3 to <24 months', 'description': 'Participants weighing 5 to less than (\\<) 7 kilogram (kg) received 25 milligram (mg) IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA administered every 12 hours (q12h) on Days 1 through 3 and 1 morning dose on Day 4.', 'interventionNames': ['Drug: IVA']}, {'type': 'EXPERIMENTAL', 'label': 'Part B + A/B:1 to < 24 months', 'description': 'Participants 4 to \\<6 months of age and weighing greater than or equal to (≥) 5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \\<7 kg received 25 mg IVA, 7 to \\<14 kg received 50 mg IVA, and those weighing 14 to \\<25 kg received 75 mg IVA q12h for 24 weeks on Part B. For Part A/B, participants 1 to \\<4 months weighing 3 kg to \\<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age.', 'interventionNames': ['Drug: IVA']}], 'interventions': [{'name': 'IVA', 'type': 'DRUG', 'otherNames': ['VX-770', 'ivacaftor'], 'description': 'Granules in sachet for oral administration.', 'armGroupLabels': ['Part A: 3 to <24 months', 'Part B + A/B:1 to < 24 months']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}, {'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'city': 'South Brisbane', 'country': 'Australia', 'geoPoint': {'lat': -27.48034, 'lon': 153.02049}}, {'city': 'Westmead', 'country': 'Australia', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'city': 'Toronto', 'country': 'Canada', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'city': 'Dublin', 'country': 'Ireland', 'geoPoint': {'lat': 53.33306, 'lon': -6.24889}}, {'city': 'Edinburgh', 'country': 'United Kingdom', 'geoPoint': {'lat': 55.95206, 'lon': -3.19648}}, {'city': 'Leicester', 'country': 'United Kingdom', 'geoPoint': {'lat': 52.6386, 'lon': -1.13169}}, {'city': 'London', 'country': 'United Kingdom', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Manchester', 'country': 'United Kingdom', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'city': 'Oxford', 'country': 'United Kingdom', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}