Ignite Creation Date:
2025-12-25 @ 1:23 AM
Ignite Modification Date:
2026-01-04 @ 8:02 AM
Study NCT ID:
NCT04537793
Status:
COMPLETED
Last Update Posted:
2023-06-28
First Post:
2020-08-28
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
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': 'C000706587', 'term': 'elexacaftor, ivacaftor, tezacaftor drug combination'}, {'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': 'Day 1 Through Safety Follow-up Visit (up to Day 43 for Part A, up to Week 28 for Part B)', 'description': 'The safety analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen.Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm.\n\nMedDRA version 23.1 applied for Part A, MedDRA version 25.0 applied for Part B.', 'eventGroups': [{'id': 'EG000', 'title': 'Part A: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.', 'otherNumAtRisk': 18, 'deathsNumAtRisk': 18, 'otherNumAffected': 15, 'seriousNumAtRisk': 18, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Part B: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing \\>=10 kg to \\<14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg qAM and IVA 59.5 mg qPM in the treatment period for 24 weeks.', 'otherNumAtRisk': 75, 'deathsNumAtRisk': 75, 'otherNumAffected': 71, 'seriousNumAtRisk': 75, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 21}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 26}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 14}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 11}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Scratch', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 8}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'SARS-CoV-2 test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 7}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 9}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Hyperamylasaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Hyperlipasaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 7}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Product taste abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 0}], 'organSystem': 'Product Issues', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 46}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 13}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 25}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Sputum increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Papule', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 12}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Rash erythematous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}], 'seriousEvents': [{'term': 'Anal incontinence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Abnormal behaviour', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}, {'term': 'Urinary incontinence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 75, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.1,25.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.'}], 'classes': [{'title': 'Day 1: ELX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Day 8: ELX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.44', 'spread': '2.09', 'groupId': 'OG000'}]}]}, {'title': 'Day 15: ELX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.69', 'spread': '2.11', 'groupId': 'OG000'}]}]}, {'title': 'Day 1: ELX-M23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Day 8: ELX-M23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.39', 'spread': '1.65', 'groupId': 'OG000'}]}]}, {'title': 'Day 15: ELX-M23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.47', 'spread': '1.65', 'groupId': 'OG000'}]}]}, {'title': 'Day 1: TEZ', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Day 8: TEZ', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.86', 'spread': '1.07', 'groupId': 'OG000'}]}]}, {'title': 'Day 15: TEZ', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.85', 'spread': '1.10', 'groupId': 'OG000'}]}]}, {'title': 'Day 1: TEZ-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Day 8: TEZ-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.42', 'spread': '1.42', 'groupId': 'OG000'}]}]}, {'title': 'Day 15: TEZ-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.68', 'spread': '1.52', 'groupId': 'OG000'}]}]}, {'title': 'Day 1: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Day 8: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.702', 'spread': '0.503', 'groupId': 'OG000'}]}]}, {'title': 'Day 15: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.746', 'spread': '0.526', 'groupId': 'OG000'}]}]}, {'title': 'Day 1: IVA-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.00', 'groupId': 'OG000'}]}]}, {'title': 'Day 8: IVA-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.78', 'spread': '0.990', 'groupId': 'OG000'}]}]}, {'title': 'Day 15: IVA-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.91', 'spread': '0.990', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Day 1 through Day 15', 'unitOfMeasure': 'micrograms per milliliter (mcg/ml)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug. Here the "Number Analyzed" signifies those participants who were evaluable at specified time point.'}, {'type': 'PRIMARY', 'title': 'Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part A: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days.'}], 'classes': [{'title': 'Participants with TEAEs', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'Participants with SAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Day 1 up to Day 43', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety set for Part A included all participants who received at least 1 dose of study drug in Part A. This outcome measure was planned only for Part A arm.'}, {'type': 'PRIMARY', 'title': 'Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '75', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg at screening received ELX 100mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \\>=10 kg to \\<14 kg received ELX 80mg qd/TEZ 40mg qd/IVA 60 mg qAM and IVA 59.5 mg qPM in the treatment period for 24 weeks.'}], 'classes': [{'title': 'Participants with TEAEs', 'categories': [{'measurements': [{'value': '74', 'groupId': 'OG000'}]}]}, {'title': 'Participants with SAEs', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Day 1 up to Week 28', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety set for Part B included all participants who received at least 1 dose of study drug in Part B. The safety and tolerability analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm.'}, {'type': 'SECONDARY', 'title': 'Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: ELX/TEZ/IVA (≥10 kg to <14 kg)', 'description': 'Participants weighing \\>=10 kg to l \\<14 kg received ELX80mg qd/TEZ 40mg qd/IVA 60 mg qAM and IVA 59.5 qPM in the treatment period for 24 weeks.'}, {'id': 'OG001', 'title': 'Part B: ELX/TEZ/IVA (≥14 kg)', 'description': 'Participants weighing \\>=14 kg at screening received ELX100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 24 weeks.'}], 'classes': [{'title': 'Day 15: ELX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '54', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2.91', 'spread': '1.38', 'groupId': 'OG000'}, {'value': '3.87', 'spread': '2.75', 'groupId': 'OG001'}]}]}, {'title': 'Week 4: ELX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '3.13', 'spread': '1.90', 'groupId': 'OG000'}, {'value': '3.56', 'spread': '2.19', 'groupId': 'OG001'}]}]}, {'title': 'Week 12: ELX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2.48', 'spread': '1.65', 'groupId': 'OG000'}, {'value': '3.49', 'spread': '2.22', 'groupId': 'OG001'}]}]}, {'title': 'Week 16: ELX', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '58', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '3.01', 'spread': '1.11', 'groupId': 'OG000'}, {'value': '3.18', 'spread': '2.19', 'groupId': 'OG001'}]}]}, {'title': 'Day 15: ELX-M23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '54', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.85', 'spread': '1.12', 'groupId': 'OG000'}, {'value': '2.33', 'spread': '1.87', 'groupId': 'OG001'}]}]}, {'title': 'Week 4: ELX-M23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '2.32', 'spread': '2.01', 'groupId': 'OG000'}, {'value': '2.22', 'spread': '1.66', 'groupId': 'OG001'}]}]}, {'title': 'Week 12: ELX-M23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.48', 'spread': '1.27', 'groupId': 'OG000'}, {'value': '2.17', 'spread': '1.69', 'groupId': 'OG001'}]}]}, {'title': 'Week 16: ELX-M23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '58', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.73', 'spread': '0.828', 'groupId': 'OG000'}, {'value': '1.94', 'spread': '1.58', 'groupId': 'OG001'}]}]}, {'title': 'Day 15: TEZ', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '52', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.73', 'spread': '0.666', 'groupId': 'OG000'}, {'value': '2.14', 'spread': '1.72', 'groupId': 'OG001'}]}]}, {'title': 'Week 4: TEZ', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.62', 'spread': '0.716', 'groupId': 'OG000'}, {'value': '1.79', 'spread': '0.947', 'groupId': 'OG001'}]}]}, {'title': 'Week 12: TEZ', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.60', 'spread': '0.951', 'groupId': 'OG000'}, {'value': '1.76', 'spread': '1.10', 'groupId': 'OG001'}]}]}, {'title': 'Week 16: TEZ', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '58', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.52', 'spread': '0.701', 'groupId': 'OG000'}, {'value': '1.80', 'spread': '1.42', 'groupId': 'OG001'}]}]}, {'title': 'Day 15: TEZ-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '52', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '7.07', 'spread': '1.08', 'groupId': 'OG000'}, {'value': '7.07', 'spread': '2.11', 'groupId': 'OG001'}]}]}, {'title': 'Week 4: TEZ-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '6.80', 'spread': '1.64', 'groupId': 'OG000'}, {'value': '7.09', 'spread': '2.18', 'groupId': 'OG001'}]}]}, {'title': 'Week 12: TEZ-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '7.06', 'spread': '2.37', 'groupId': 'OG000'}, {'value': '7.18', 'spread': '2.38', 'groupId': 'OG001'}]}]}, {'title': 'Week 16: TEZ-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '58', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '6.88', 'spread': '1.94', 'groupId': 'OG000'}, {'value': '6.95', 'spread': '2.77', 'groupId': 'OG001'}]}]}, {'title': 'Day 15: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '52', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.549', 'spread': '0.373', 'groupId': 'OG000'}, {'value': '0.661', 'spread': '0.552', 'groupId': 'OG001'}]}]}, {'title': 'Week 4: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.535', 'spread': '0.402', 'groupId': 'OG000'}, {'value': '0.594', 'spread': '0.405', 'groupId': 'OG001'}]}]}, {'title': 'Week 12: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.383', 'spread': '0.203', 'groupId': 'OG000'}, {'value': '0.530', 'spread': '0.474', 'groupId': 'OG001'}]}]}, {'title': 'Week 16: IVA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '58', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '0.483', 'spread': '0.347', 'groupId': 'OG000'}, {'value': '0.580', 'spread': '0.511', 'groupId': 'OG001'}]}]}, {'title': 'Day 15: IVA-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '52', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.47', 'spread': '0.969', 'groupId': 'OG000'}, {'value': '1.67', 'spread': '0.961', 'groupId': 'OG001'}]}]}, {'title': 'Week 4: IVA-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.64', 'spread': '1.29', 'groupId': 'OG000'}, {'value': '1.52', 'spread': '0.737', 'groupId': 'OG001'}]}]}, {'title': 'Week 12: IVA-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.19', 'spread': '0.602', 'groupId': 'OG000'}, {'value': '1.45', 'spread': '0.907', 'groupId': 'OG001'}]}]}, {'title': 'Week 16: IVA-M1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '58', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '1.24', 'spread': '0.673', 'groupId': 'OG000'}, {'value': '1.51', 'spread': '0.911', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Day 1 through Week 16', 'unitOfMeasure': 'micrograms per milliliter (mcg/ml)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set included participants who received at least 1 dose of study drug. Here the "Number Analyzed" signifies those participants who were evaluable at specified time point.'}, {'type': 'SECONDARY', 'title': 'Part B: Absolute Change in Sweat Chloride (SwCl)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '75', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg received ELX 100mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \\>=10 kg to \\<14 kg received ELX 80mg qd/TEZ 40mg qd/IVA 60 mg qAM and IVA 59.5mg qPM in the treatment period for 24 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-57.9', 'groupId': 'OG000', 'lowerLimit': '-61.3', 'upperLimit': '-54.6'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline through Week 24', 'description': 'Sweat samples were collected using an approved collection device.', 'unitOfMeasure': 'millimole per liter (mmol/L)', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm.'}, {'type': 'SECONDARY', 'title': 'Part B: Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part B: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg received ELX 100 mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \\>=10 kg to less than \\<14 kg received ELX 80 mg qd/TEZ 40mg qd/IVA 60 mg qAM and IVA 59.5 mg qPM in the treatment period for 24 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.83', 'groupId': 'OG000', 'lowerLimit': '-1.01', 'upperLimit': '-0.66'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline through Week 24', 'description': 'The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.', 'unitOfMeasure': 'index', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Part A: ELX/TEZ/IVA', 'description': 'Participants weighing greater than or equals to (\\>=)14 kg at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days.'}, {'id': 'FG001', 'title': 'Part B: ELX/TEZ/IVA', 'description': 'Participants weighing \\>=14 kg at screening received ELX 100mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \\>=10 kg to less than (\\<)14 kg received ELX 80mg qd/TEZ 40mg qd/IVA 60 mg once every morning (qAM) and IVA 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.'}], 'periods': [{'title': 'Part A (15 Days)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '18'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'Part B (24 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'comment': 'Out of the 18 participants from Part A, 10 participants also participated in Part B of the study.', 'groupId': 'FG001', 'numSubjects': '75'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '74'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'The study was conducted in 2 parts, Part A and Part B. The Participant flow was planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen.', 'preAssignmentDetails': 'This study was conducted in participants with cystic fibrosis (CF) aged 2 through 5 years of age (inclusive).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '83', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'ELX/TEZ/IVA', 'description': 'Part A: Participants weighing greater than or equal to (\\>=)14 kilograms (kg) at screening received ELX 100 milligrams (mg) once daily (qd)/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) in the treatment period for 15 days.\n\nPart B: Participants weighing \\>=14 kg at screening received ELX 100mg qd/TEZ 50mg qd/IVA 75 mg q12h and participants weighing \\>=10 kg to less than (\\<)14 kg received ELX 80mg qd/TEZ 40mg qd/IVA 60 mg once every morning (qAM) and IVA 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'title': 'Part A', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'BG000'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '18', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Part B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '75', 'groupId': 'BG000'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '75', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. 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': '18', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}, {'title': 'Part B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '75', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '41', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '34', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. 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': '18', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '18', 'groupId': 'BG000'}]}, {'title': 'Not collected per local regulations', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Part B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '75', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '63', 'groupId': 'BG000'}]}, {'title': 'Not collected per local regulations', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. 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': '18', 'groupId': 'BG000'}]}], 'categories': [{'title': 'White', 'measurements': [{'value': '18', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not collected per local regulations', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}, {'title': 'Part B', 'denoms': [{'units': 'Participants', 'counts': [{'value': '75', 'groupId': 'BG000'}]}], 'categories': [{'title': 'White', 'measurements': [{'value': '68', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not collected per local regulations', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'The Baseline data were planned to be presented for the overall treatment arms (i.e. Part A and Part B), irrespective of weight-based dose regimen. 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': '2021-10-21', 'size': 5295060, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-05-30T06:58', 'hasProtocol': True}, {'date': '2022-05-02', 'size': 4414223, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-05-30T06:59', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 83}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-11-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-06', 'completionDateStruct': {'date': '2022-06-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-06-02', 'studyFirstSubmitDate': '2020-08-28', 'resultsFirstSubmitDate': '2023-06-02', 'studyFirstSubmitQcDate': '2020-08-28', 'lastUpdatePostDateStruct': {'date': '2023-06-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-06-02', 'studyFirstPostDateStruct': {'date': '2020-09-03', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-06-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-06-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites', 'timeFrame': 'From Day 1 through Day 15'}, {'measure': 'Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'From Day 1 up to Day 43'}, {'measure': 'Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'From Day 1 up to Week 28'}], 'secondaryOutcomes': [{'measure': 'Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites', 'timeFrame': 'From Day 1 through Week 16'}, {'measure': 'Part B: Absolute Change in Sweat Chloride (SwCl)', 'timeFrame': 'From Baseline through Week 24', 'description': 'Sweat samples were collected using an approved collection device.'}, {'measure': 'Part B: Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)', 'timeFrame': 'From Baseline through Week 24', 'description': 'The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '37983082', 'type': 'DERIVED', 'citation': 'Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.'}, {'pmid': '36921081', 'type': 'DERIVED', 'citation': 'Goralski JL, Hoppe JE, Mall MA, McColley SA, McKone E, Ramsey B, Rayment JH, Robinson P, Stehling F, Taylor-Cousar JL, Tullis E, Ahluwalia N, Chin A, Chu C, Lu M, Niu T, Weinstock T, Ratjen F, Rosenfeld M. Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2023 Jul 1;208(1):59-67. doi: 10.1164/rccm.202301-0084OC.'}, {'pmid': '33331662', 'type': 'DERIVED', 'citation': 'Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.'}]}, 'descriptionModule': {'briefSummary': 'This study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination therapy in CF subjects 2 through 5 years of age.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '5 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Homozygous for the F508del mutation or heterozygous for F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)\n\nKey Exclusion Criteria:\n\n* Clinically significant cirrhosis with or without portal hypertension\n* Lung infection with organisms associated with a more rapid decline in pulmonary status\n* Solid organ or hematological transplantation\n\nOther protocol defined Inclusion/Exclusion criteria may apply'}, 'identificationModule': {'nctId': 'NCT04537793', 'briefTitle': 'Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Through 5 Years of Age', 'orgStudyIdInfo': {'id': 'VX20-445-111'}, 'secondaryIdInfos': [{'id': '2020-002251-38', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part A: ELX/TEZ/IVA', 'description': 'Participants weighing greater than or equal to (\\>=)14 kilograms (kg) at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days.', 'interventionNames': ['Drug: ELX/TEZ/IVA', 'Drug: IVA']}, {'type': 'EXPERIMENTAL', 'label': 'Part B: ELX/TEZ/IVA', 'description': 'Participants weighing (\\>=)14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h. Participants weighing (\\>=)10 kg to less than (\\<)14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg once every morning (qAM) and 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.', 'interventionNames': ['Drug: ELX/TEZ/IVA', 'Drug: IVA']}], 'interventions': [{'name': 'ELX/TEZ/IVA', 'type': 'DRUG', 'otherNames': ['VX-445/VX-661/VX-770', 'elexacaftor/tezacaftor/ivacaftor'], 'description': 'Fixed dose combination granules for oral administration.', 'armGroupLabels': ['Part A: ELX/TEZ/IVA', 'Part B: ELX/TEZ/IVA']}, {'name': 'IVA', 'type': 'DRUG', 'otherNames': ['VX-770', 'ivacaftor'], 'description': 'Granules for oral administration.', 'armGroupLabels': ['Part A: ELX/TEZ/IVA', 'Part B: ELX/TEZ/IVA']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85724', 'city': 'Tucson', 'state': 'Arizona', 'country': 'United States', 'facility': 'Banner University of Arizona Medical Center', 'geoPoint': {'lat': 32.22174, 'lon': -110.92648}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital of Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H. 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