Ignite Creation Date:
2025-12-25 @ 5:11 AM
Ignite Modification Date:
2025-12-26 @ 4:16 AM
Study NCT ID:
NCT00909727
Status:
COMPLETED
Last Update Posted:
2012-08-21
First Post:
2009-05-26
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}, {'id': 'D005355', 'term': 'Fibrosis'}, {'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': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D010335', 'term': 'Pathologic Processes'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C545203', 'term': 'ivacaftor'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medicalinfo@vrtx.com', 'phone': '617-444-6777', 'title': 'Medical Monitor', 'organization': 'Vertex'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'For enrolled subjects, adverse events (AEs) were collected through the Follow-up visit in each study part. For subjects who completed 48 weeks of treatment and enrolled in the open-label extension study, AEs were only collected through the Week 48 visit.', 'eventGroups': [{'id': 'EG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks', 'otherNumAtRisk': 26, 'otherNumAffected': 25, 'seriousNumAtRisk': 26, 'seriousNumAffected': 6}, {'id': 'EG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks', 'otherNumAtRisk': 26, 'otherNumAffected': 26, 'seriousNumAtRisk': 26, 'seriousNumAffected': 5}], 'otherEvents': [{'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 40, 'numAffected': 19}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 21, 'numAffected': 13}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 9, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 10, 'numAffected': 7}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 7, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 8, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rales', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pharyngeal erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Respiratory tract congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rhinitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 9, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 9, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 11, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 5, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 6, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Bacteria sputum identified', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pulmonary function test decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Eosinophil count increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Culture throat positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Breath sounds abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Forced expiratory volume decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Neutrophil count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'White blood cell count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 16, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Excoriation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Cystic fibrosis lung', 'notes': 'CF exacerbations were coded as "cystic fibrosis lung."', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 8, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 8, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 15, 'numAffected': 7}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Lymphadenopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}], 'seriousEvents': [{'term': 'Cystic fibrosis lung', 'notes': 'CF exacerbations were coded as "cystic fibrosis lung."', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Lung consolidation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pulmonary function test decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Adjustment disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Affective disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Conversion disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Pseudomonas infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}, {'term': 'Muscle strain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 26, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 26, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (12.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks'}], 'classes': [{'categories': [{'measurements': [{'value': '0.1', 'spread': '2.1', 'groupId': 'OG000'}, {'value': '12.6', 'spread': '2.1', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '12.5', 'ciLowerLimit': '6.6', 'ciUpperLimit': '18.3', 'pValueComment': "The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '2.9', 'groupDescription': 'The primary analysis for the primary efficacy variable was based on a Mixed-Effects Model for Repeated Measures (MMRM). The model included absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) as the dependent variable, treatment (ivacaftor versus placebo) and visit (Day 15, Week 8, Week 16, and Week 24) as fixed effects, and subject as a random effect, with adjustment for the continuous baseline value of percent predicted FEV1.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': 'Denominator degrees of freedom were estimated using the Kenward-Roger approximation. No imputation of missing data was done.', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 24 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.', 'unitOfMeasure': 'percent of predicted volume (L)', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo)and had available assessments during the time frame.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks'}], 'classes': [{'categories': [{'measurements': [{'value': '0.7', 'spread': '2.0', 'groupId': 'OG000'}, {'value': '10.7', 'spread': '1.9', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0006', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '10.0', 'ciLowerLimit': '4.5', 'ciUpperLimit': '15.5', 'pValueComment': 'There was no adjustment for multiple comparisons.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '2.7', 'groupDescription': 'Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint, a Mixed-Effects Model for Repeated Measures (MMRM). Estimates were obtained from MMRM with dependent variable absolute change from baseline, fixed effects for categorical visit \\& treatment group, \\& adjustment for the continuous baseline value of percent predicted FEV1, using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': 'Denominator degrees of freedom were estimated using the Kenward-Roger approximation. no imputation of missing data was done.', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 48 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.', 'unitOfMeasure': 'percent of predicted volume (L)', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Through Week 24 and Week 48 (Respiratory Domain Score, Children)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks'}], 'classes': [{'title': 'Change from Baseline Through Week 24', 'categories': [{'measurements': [{'value': '0.3', 'spread': '2.6', 'groupId': 'OG000'}, {'value': '6.3', 'spread': '2.5', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline Through Week 48', 'categories': [{'measurements': [{'value': '1.0', 'spread': '2.3', 'groupId': 'OG000'}, {'value': '6.1', 'spread': '2.2', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.1092', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '6.1', 'ciLowerLimit': '-1.4', 'ciUpperLimit': '13.5', 'pValueComment': "The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '3.7', 'groupDescription': 'Through Week 24: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint, a Mixed-Effects Model for Repeated Measures (MMRM), with the addition of the baseline domain score as a covariate.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '0.1354', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '5.1', 'ciLowerLimit': '-1.6', 'ciUpperLimit': '11.8', 'pValueComment': 'There was no adjustment for multiple comparisons.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '3.3', 'groupDescription': 'Through Week 48: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint, a Mixed-Effects Model for Repeated Measures (MMRM), with the addition of the baseline domain score as a covariate.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}, {'value': '23', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks'}], 'classes': [{'title': 'Change from Baseline Through Week 24', 'categories': [{'measurements': [{'value': '-1.2', 'spread': '2.6', 'groupId': 'OG000'}, {'value': '-55.5', 'spread': '2.6', 'groupId': 'OG001'}]}]}, {'title': 'Change from Baseline Through Week 48', 'categories': [{'measurements': [{'value': '-2.6', 'spread': '2.6', 'groupId': 'OG000'}, {'value': '-56.0', 'spread': '2.5', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-54.3', 'ciLowerLimit': '-61.8', 'ciUpperLimit': '-46.8', 'pValueComment': "The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '3.7', 'groupDescription': 'Through Week 24: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for sweat chloride and percent predicted forced expiratory volume in 1 second (FEV1), using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-53.5', 'ciLowerLimit': '-60.9', 'ciUpperLimit': '-46.0', 'pValueComment': 'There was no adjustment for multiple comparisons.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '3.7', 'groupDescription': 'Through Week 48: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for sweat chloride and percent predicted forced expiratory volume in 1 second (FEV1), using unstructured covariance matrix.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.', 'unitOfMeasure': 'millimoles per liter', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Weight at Week 24 and Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks'}, {'id': 'OG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks'}], 'classes': [{'title': 'At Week 24', 'categories': [{'measurements': [{'value': '1.8', 'spread': '0.4', 'groupId': 'OG000'}, {'value': '3.7', 'spread': '0.4', 'groupId': 'OG001'}]}]}, {'title': 'At Week 48', 'categories': [{'measurements': [{'value': '3.1', 'spread': '0.5', 'groupId': 'OG000'}, {'value': '5.9', 'spread': '0.5', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0004', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.9', 'ciLowerLimit': '0.9', 'ciUpperLimit': '2.9', 'pValueComment': "The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).", 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.5', 'groupDescription': 'At Week 24: Analysis for this variable was based on a Linear Mixed Effect (LME) model with dependent variable weight; treatment as a fixed effect; and intercept, visit, and treatment by visit interaction as random effects, with adjustment for baseline percent predicted forced expiratory volume in 1 second (FEV1) severity.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '0.0002', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.8', 'ciLowerLimit': '1.3', 'ciUpperLimit': '4.2', 'pValueComment': 'P-value is for the treatment effect at Week 48 (obtained as a linear contrast of treatment at Day 336). There was no adjustment for multiple comparisons.', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.7', 'groupDescription': 'At Week 48: Analysis for this variable was based on a Linear Mixed Effect (LME) model with random intercept and random slope, treatment as a fixed effect, and visit (days on study) and treatment by visit interaction as random effects, with adjustment for categorical baseline percent predicted forced expiratory volume in 1 second (FEV1) severity.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'baseline to 24 weeks and 48 weeks', 'description': 'As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.', 'unitOfMeasure': 'kilograms', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks'}, {'id': 'FG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'All subjects who received at least 1 dose of study drug (placebo)', 'groupId': 'FG000', 'numSubjects': '26'}, {'comment': 'All subjects who received at least 1 dose of study drug (ivacaftor)', 'groupId': 'FG001', 'numSubjects': '26'}]}, {'type': 'Completed Treatment Period, Week 24', 'achievements': [{'groupId': 'FG000', 'numSubjects': '23'}, {'groupId': 'FG001', 'numSubjects': '26'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Completed Treatment and Extension Periods (through Week 48)', 'groupId': 'FG000', 'numSubjects': '22'}, {'comment': 'Completed Treatment and Extension Periods (through Week 48)', 'groupId': 'FG001', 'numSubjects': '26'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Wrong Genotype', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Withdrawal of Consent', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Prohibited Medication', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Part A started on 05 August 2009 (signing of first informed consent). Screening evaluations were completed during Day -28 to Day -2. All subjects completing Part A were offered the opportunity to participate in Part B, which started on 12 March 2010. Screening evaluations were completed during Day -35 to Day -15 before the first dose of study drug.', 'preAssignmentDetails': 'Nine subjects were dosed and included in Part A. In Part B, 52 subjects were enrolled and all were randomized to ivacaftor (26 subjects) or placebo (26 subjects). A 2-week run-in period was included to establish the baseline assessments on Day 1 after ensuring that subjects were properly taking their cystic fibrosis (CF) medication regimens.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'BG000'}, {'value': '26', 'groupId': 'BG001'}, {'value': '52', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Placebo', 'description': 'Oral tablet every 12 hours (q12h) for up to 48 weeks'}, {'id': 'BG001', 'title': '150 mg Ivacaftor q12h', 'description': 'Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age Continuous', 'classes': [{'categories': [{'measurements': [{'value': '8.9', 'spread': '1.86', 'groupId': 'BG000'}, {'value': '8.9', 'spread': '2.00', 'groupId': 'BG001'}, {'value': '8.9', 'spread': '1.91', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Age, Customized', 'classes': [{'title': '6 to 8 Years', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}]}]}, {'title': '9 to 11 Years', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}]}, {'title': '> 11 Years', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '22', 'groupId': 'BG001'}, {'value': '45', 'groupId': 'BG002'}]}]}, {'title': 'Other', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}, {'title': 'Not Allowed to Ask Per Local Regulations', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Hispanic or Latino', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '24', 'groupId': 'BG000'}, {'value': '23', 'groupId': 'BG001'}, {'value': '47', 'groupId': 'BG002'}]}]}, {'title': 'Not Allowed to Ask Per Local Regulations', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'North America', 'categories': [{'measurements': [{'value': '15', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}]}, {'title': 'Europe', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}, {'title': 'Australia', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Weight', 'classes': [{'categories': [{'measurements': [{'value': '30.0', 'spread': '7.16', 'groupId': 'BG000'}, {'value': '31.8', 'spread': '9.95', 'groupId': 'BG001'}, {'value': '30.9', 'spread': '8.63', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kilograms', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Body Mass Index', 'classes': [{'categories': [{'measurements': [{'value': '16.8', 'spread': '1.75', 'groupId': 'BG000'}, {'value': '17.1', 'spread': '2.61', 'groupId': 'BG001'}, {'value': '17.0', 'spread': '2.21', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kilograms per square meter', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sweat Chloride', 'classes': [{'categories': [{'measurements': [{'value': '104.8', 'spread': '8.87', 'groupId': 'BG000'}, {'value': '104.3', 'spread': '14.54', 'groupId': 'BG001'}, {'value': '104.6', 'spread': '11.92', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'millimoles per liter', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)', 'classes': [{'title': '< 70%', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}, {'title': '≥ 70% to ≤ 90%', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}]}, {'title': '> 90%', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '22', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'description': 'Percent predicted for age, gender, and height', 'unitOfMeasure': 'participants'}, {'title': 'Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)', 'classes': [{'categories': [{'measurements': [{'value': '83.7', 'spread': '20.37', 'groupId': 'BG000'}, {'value': '84.7', 'spread': '15.83', 'groupId': 'BG001'}, {'value': '84.2', 'spread': '18.07', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'Percent predicted for age, gender, and height', 'unitOfMeasure': 'percentage', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 52}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2012-07', 'dispFirstSubmitDate': '2012-01-03', 'completionDateStruct': {'date': '2011-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2012-07-18', 'studyFirstSubmitDate': '2009-05-26', 'dispFirstSubmitQcDate': '2012-01-03', 'resultsFirstSubmitDate': '2012-02-27', 'studyFirstSubmitQcDate': '2009-05-26', 'dispFirstPostDateStruct': {'date': '2012-01-04', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2012-08-21', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2012-07-18', 'studyFirstPostDateStruct': {'date': '2009-05-28', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2012-08-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24', 'timeFrame': 'baseline through 24 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.'}], 'secondaryOutcomes': [{'measure': 'Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 48', 'timeFrame': 'baseline through 48 weeks', 'description': 'Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.'}, {'measure': 'Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Through Week 24 and Week 48 (Respiratory Domain Score, Children)', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).'}, {'measure': 'Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48', 'timeFrame': 'baseline through 24 weeks and 48 weeks', 'description': 'The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.'}, {'measure': 'Absolute Change From Baseline in Weight at Week 24 and Week 48', 'timeFrame': 'baseline to 24 weeks and 48 weeks', 'description': 'As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Fibrosis', 'Pancreatic Diseases', 'Digestive System Diseases', 'Lung Diseases', 'Respiratory Tract Diseases', 'Genetic Diseases, Inborn', 'Infant, Newborn, Diseases', 'Pathologic Processes'], 'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '23590265', 'type': 'DERIVED', 'citation': 'Davies JC, Wainwright CE, Canny GJ, Chilvers MA, Howenstine MS, Munck A, Mainz JG, Rodriguez S, Li H, Yen K, Ordonez CL, Ahrens R; VX08-770-103 (ENVISION) Study Group. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013 Jun 1;187(11):1219-25. doi: 10.1164/rccm.201301-0153OC.'}], 'seeAlsoLinks': [{'url': 'http://ghr.nlm.nih.gov/', 'label': 'Genetics Home Reference'}, {'url': 'http://www.nlm.nih.gov/medlineplus/', 'label': 'Medline Plus'}, {'url': 'http://www.clinicaltrials.gov/ct2/info/fdalinks', 'label': 'U.S. FDA Resources'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic adenosine monophosphate (AMP)-dependent protein kinase A (PKA) activation.', 'detailedDescription': 'This is a Phase 3, 2-part, randomized, double-blind, placebo-controlled, parallel group multicenter study of orally administered ivacaftor in subjects with cystic fibrosis (CF) 6 to 11 years of age who have the G551D-CFTR mutation and a forced expiratory volume in 1 second (FEV1) between 90% and 105% predicted (using Knudson standards).\n\nBased on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating effect of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.\n\nThis study was conducted in 2 parts. Part A was conducted to analyze the PK properties of ivacaftor and to determine the most appropriate dose to administer to subjects in Part B of this study. Part B explored the safety and efficacy of ivacaftor over long-term treatment in subjects 6 to 11 years of age.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Weighing at least 15 kg\n* Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele\n* Forced expiratory volume in 1 second (FEV1) of 40% to 105% (inclusive) of predicted normal for age, gender, and height (Knudson standards) at Screening\n* Able to swallow tablets\n* As judged by the investigator, parent or legal guardian and subject must have been able to understand protocol requirements, restrictions, and instructions, and the parent or legal guardian should have been able to ensure that the subject complied with, and was likely to complete, the study as planned\n* Parent or legal guardian must have signed the informed consent form and corresponding assent must be obtained from the subject\n* Willing to use at least 1 highly effective birth control method during the study\n* No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator\n\nExclusion Criteria:\n\n* History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject\n* Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study\n* Abnormal liver function ≥ 3x the upper limit of normal\n* Abnormal renal function at Screening\n* History of solid organ or hematological transplantation\n* Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening\n* Use of inhaled hypertonic saline treatment\n* Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)'}, 'identificationModule': {'nctId': 'NCT00909727', 'acronym': 'ENVISION', 'briefTitle': 'Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX-770 in Subjects Aged 6 to 11 Years With Cystic Fibrosis and the G551D Mutation', 'orgStudyIdInfo': {'id': 'VX08-770-103'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Subjects who received placebo every 12 hours (q12h) for up to 48 weeks.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': '150 mg Ivacaftor q12h', 'description': 'Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks.', 'interventionNames': ['Drug: Ivacaftor']}], 'interventions': [{'name': 'Ivacaftor', 'type': 'DRUG', 'otherNames': ['VX-770'], 'description': '150-mg tablet given orally q12h for up to 48 weeks', 'armGroupLabels': ['150 mg Ivacaftor q12h']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Tablet given orally q12h for up to 48 weeks', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233-1711', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory Cystic Fibrosis Center', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60614', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Children's Memorial Hospital", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '60025', 'city': 'Glenview', 'state': 'Illinois', 'country': 'United States', 'facility': 'The Cystic Fibrosis 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'48201', 'city': 'Detroit', 'state': 'Michigan', 'country': 'United States', 'facility': "Children's Hospital of Michigan", 'geoPoint': {'lat': 42.33143, 'lon': -83.04575}}, {'zip': '49503', 'city': 'Grand Rapids', 'state': 'Michigan', 'country': 'United States', 'facility': "Helen DeVos Children's Hospital Spectrum Health Hospitals", 'geoPoint': {'lat': 42.96336, 'lon': -85.66809}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'facility': "The Children's Mercy Hospital", 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '68195-5190', 'city': 'Omaha', 'state': 'Nebraska', 'country': 'United States', 'facility': 'University of Nebraska Medical Center Pediatric Pulmonary/ CF', 'geoPoint': {'lat': 41.25626, 'lon': -95.94043}}, {'zip': '37916', 'city': 'Knoxville', 'state': 'Tennessee', 'country': 'United States', 'facility': "East Tennessee Children's Hospital Pediatric Pulmonary and Respiratory Care", 'geoPoint': {'lat': 35.96064, 'lon': -83.92074}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Pediatric Pulmonology', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '22908', 'city': 'Charlottesville', 'state': 'Virginia', 'country': 'United States', 'facility': 'University of Virginia Pediatric Respiratory Medicine', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}, {'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': "The Children's Hospital Westmead", 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '4029', 'city': 'Herston', 'state': 'Queensland', 'country': 'Australia', 'facility': "Royal Children's Hospital Brisbane", 'geoPoint': {'lat': -27.44453, 'lon': 153.01852}}, {'zip': '3052', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': "Royal Children's Hospital Melbourne", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': '6008', 'city': 'Subiaco', 'state': 'Western Australia', 'country': 'Australia', 'facility': 'Princess Margaret Hospital for Children', 'geoPoint': {'lat': -31.9485, 'lon': 115.8268}}, {'zip': 'V6H-3V4', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "British Columbia Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Hospital for Sick Children CF Center', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': '75935', 'city': 'Paris', 'country': 'France', 'facility': 'Hôpital Robert Debré - Service de gastro-entérologiemucoviscidose et nutrition', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '91054', 'city': 'Erlangen', 'country': 'Germany', 'facility': 'Kinder- und Jugendklinik Universitätsklinikum Erlangen', 'geoPoint': {'lat': 49.59099, 'lon': 11.00783}}, {'zip': '07740', 'city': 'Jena', 'country': 'Germany', 'facility': 'Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin', 'geoPoint': {'lat': 50.92878, 'lon': 11.5899}}, {'zip': '12', 'city': 'Dublin', 'country': 'Ireland', 'facility': "Our Lady's Children's Hospital", 'geoPoint': {'lat': 53.33306, 'lon': -6.24889}}, {'zip': '24', 'city': 'Dublin', 'country': 'Ireland', 'facility': "The National Children's Hospital", 'geoPoint': {'lat': 53.33306, 'lon': -6.24889}}, {'zip': 'SW3 6LR', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Dept of Gene Therapy, Imperial College London', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Richard Ahrens, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Roy A. & Lucille A. Carver College of Medicine'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Cystic Fibrosis Foundation', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}