Ignite Creation Date:
2025-12-24 @ 1:33 PM
Ignite Modification Date:
2026-02-23 @ 6:27 AM
Study NCT ID:
NCT03125395
Status:
COMPLETED
Last Update Posted:
2020-08-07
First Post:
2017-04-19
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000599212', 'term': 'lumacaftor, ivacaftor drug combination'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medicalinfo@vrtx.com', 'phone': '617-341-6777', 'title': 'Medical Monitor', 'organization': 'Vertex Pharmaceuticals Incorporated'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Day 1 up to Week 98', 'eventGroups': [{'id': 'EG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.', 'otherNumAtRisk': 57, 'deathsNumAtRisk': 57, 'otherNumAffected': 55, 'seriousNumAtRisk': 57, 'deathsNumAffected': 0, 'seriousNumAffected': 15}], 'otherEvents': [{'term': 'Ear pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 17}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 23}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Vessel puncture site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 12}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hand-foot-and-mouth disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 10}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 12}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 13}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 10}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 5}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Forced expiratory volume decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pseudomonas test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 9}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Staphylococcus test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 12}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 6}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 47}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Lower respiratory tract congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 25}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Nasal discharge discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 10}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Productive cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 18}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Sinus congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Sputum increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Upper respiratory tract congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}], 'seriousEvents': [{'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Haematemesis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Pancreatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Chronic sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Gastritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Gastroenteritis adenovirus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Weight gain poor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}, {'term': 'Sleep apnoea syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 57, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Meddra 22.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'title': 'Participants with AEs', 'categories': [{'measurements': [{'value': '56', 'groupId': 'OG000'}]}]}, {'title': 'Participants with SAEs', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 1 up to Week 98', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 safety set included all participants dosed in study 115B who were exposed to any amount of study drug in current study 116 treatment cohort.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Sweat Chloride', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '-29.6', 'spread': '12.7', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'Sweat samples were collected using an approved collection device.', 'unitOfMeasure': 'millimole per liter (mmol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 Full Analysis Set (FAS) included all participants who were enrolled and exposed to any amount of study drug in current study 116.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Body Mass Index (BMI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.30', 'spread': '1.21', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'BMI was defined as weight in kilogram (kg) divided by height in square meter (m\\^2).', 'unitOfMeasure': 'kg/m^2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in BMI-for-age Z-score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.27', 'spread': '0.73', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'BMI was defined as weight in kilograms divided by height in m\\^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.', 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Weight', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '6.0', 'spread': '2.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'unitOfMeasure': 'kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Weight-for-age Z-score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.23', 'spread': '0.56', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'Z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.', 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Stature (Height)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '16.1', 'spread': '2.3', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'unitOfMeasure': 'centimeter', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Stature-for-age Z-score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.07', 'spread': '0.39', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'Z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.', 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Time-to-first Pulmonary Exacerbation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '600.0', 'comment': 'Upper limit of inter-quartile range could not be estimated because less than 75% of participants had events.', 'groupId': 'OG000', 'lowerLimit': '98.0', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'From Parent Study 115B Baseline through Week 96', 'description': 'Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol-defined criteria.', 'unitOfMeasure': 'days', 'dispersionType': 'Inter-Quartile Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 115B FAS included all participants who were enrolled and exposed to any amount of study drug in parent study 115B'}, {'type': 'SECONDARY', 'title': 'Number of Pulmonary Exacerbations (PEx)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '82', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Parent Study 115B Baseline through Week 96', 'description': 'Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol-defined criteria.', 'unitOfMeasure': 'pulmonary exacerbation events', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 115B FAS.'}, {'type': 'SECONDARY', 'title': 'Number of Cystic Fibrosis (CF) Related Hospitalizations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '28', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Parent Study 115B Baseline through Week 96', 'unitOfMeasure': 'hospitalizations', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 115B FAS.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Fecal Elastase-1 (FE-1) Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '132.6', 'spread': '174.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'unitOfMeasure': 'microgram per gram (mcg/g)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Immunoreactive Trypsinogen (IRT) Serum Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '-108.5', 'spread': '306.6', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'unitOfMeasure': 'nanogram per milliliter (ng/mL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Microbiology Culture Status (Positive or Negative)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '46', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'title': 'Burkholderia (Positive)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Burkholderia (Negative)', 'categories': [{'measurements': [{'value': '46', 'groupId': 'OG000'}]}]}, {'title': 'MRSA (Positive)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'MRSA (Negative)', 'categories': [{'measurements': [{'value': '42', 'groupId': 'OG000'}]}]}, {'title': 'MSSA (Positive)', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'MSSA (Negative)', 'categories': [{'measurements': [{'value': '31', 'groupId': 'OG000'}]}]}, {'title': 'P. Aeruginosa Mucoid (Positive)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'P. Aeruginosa Mucoid (Negative)', 'categories': [{'measurements': [{'value': '46', 'groupId': 'OG000'}]}]}, {'title': 'P. Aeruginosa Non-Mucoid (Positive)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'P. Aeruginosa Non-Mucoid (Negative)', 'categories': [{'measurements': [{'value': '45', 'groupId': 'OG000'}]}]}, {'title': 'P. Aeruginosa Small Colony Variant (Positive)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'P. Aeruginosa Small Colony Variant (Negative)', 'categories': [{'measurements': [{'value': '46', 'groupId': 'OG000'}]}]}, {'title': 'Stenotrophomonas Maltophilia (Positive)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Stenotrophomonas Maltophilia (Negative)', 'categories': [{'measurements': [{'value': '44', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Week 96', 'description': 'Following microbial tests were performed: Burkholderia, Methicillin Resistant Staphylococcus Aureus (MRSA), Methicillin Susceptible Staphylococcus Aureus (MSSA), Pseudomonas Aeruginosa Mucoid (P. Aeruginosa Mucoid), P. Aeruginosa Non-Mucoid, P. Aeruginosa Small Colony Variant and Stenotrophomonas Maltophilia.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome at Week 96.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Lung Clearance Index (LCI) 2.5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.20', 'spread': '1.55', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.', 'unitOfMeasure': 'lung clearance index', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 LCI substudy set included all participants who signed the informed consent/assent to the optional LCI substudy and were enrolled and exposed to any amount of study drug in current study 116.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Lung Clearance Index (LCI) 5.0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.11', 'spread': '0.65', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.', 'unitOfMeasure': 'lung clearance index', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Study 116 LCI substudy set.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '57'}]}, {'type': 'Study 115B FAS', 'comment': 'Participants enrolled and exposed to any amount of study drug in parent study VX15-809-115.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '60'}]}, {'type': 'Study 116 FAS', 'comment': 'Participants enrolled and exposed to any amount of study drug in current study VX16-809-116.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '57'}]}, {'type': 'Study 116 LCI Substudy Set', 'achievements': [{'groupId': 'FG000', 'numSubjects': '31'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '47'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Withdrawal of consent (not due to AE)', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Commercial drug is available', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}]}], 'recruitmentDetails': 'This study was planned to have 2 cohorts: Treatment Cohort and Observational Cohort. Only the Treatment Cohort is presented in results as there were no participants enrolled in the Observational Cohort.', 'preAssignmentDetails': 'This study was conducted in participants with cystic fibrosis (CF) aged 2 years and older who participated in parent study VX15-809-115 Part B (NCT02797132).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '43.2', 'spread': '12.17', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'months', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '28', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '29', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '54', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '56', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-10-10', 'size': 935234, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-07-15T09:23', 'hasProtocol': True}, {'date': '2019-05-20', 'size': 5802549, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-07-15T09:25', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 57}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-05-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-07', 'completionDateStruct': {'date': '2019-07-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-07-15', 'studyFirstSubmitDate': '2017-04-19', 'resultsFirstSubmitDate': '2020-07-15', 'studyFirstSubmitQcDate': '2017-04-21', 'lastUpdatePostDateStruct': {'date': '2020-08-07', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-07-15', 'studyFirstPostDateStruct': {'date': '2017-04-24', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2020-08-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-07-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Day 1 up to Week 98'}], 'secondaryOutcomes': [{'measure': 'Absolute Change in Sweat Chloride', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'Sweat samples were collected using an approved collection device.'}, {'measure': 'Absolute Change in Body Mass Index (BMI)', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'BMI was defined as weight in kilogram (kg) divided by height in square meter (m\\^2).'}, {'measure': 'Absolute Change in BMI-for-age Z-score', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'BMI was defined as weight in kilograms divided by height in m\\^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.'}, {'measure': 'Absolute Change in Weight', 'timeFrame': 'From Parent Study 115B Baseline at Week 96'}, {'measure': 'Absolute Change in Weight-for-age Z-score', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'Z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.'}, {'measure': 'Absolute Change From Baseline in Stature (Height)', 'timeFrame': 'From Parent Study 115B Baseline at Week 96'}, {'measure': 'Absolute Change in Stature-for-age Z-score', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'Z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.'}, {'measure': 'Time-to-first Pulmonary Exacerbation', 'timeFrame': 'From Parent Study 115B Baseline through Week 96', 'description': 'Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol-defined criteria.'}, {'measure': 'Number of Pulmonary Exacerbations (PEx)', 'timeFrame': 'From Parent Study 115B Baseline through Week 96', 'description': 'Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol-defined criteria.'}, {'measure': 'Number of Cystic Fibrosis (CF) Related Hospitalizations', 'timeFrame': 'From Parent Study 115B Baseline through Week 96'}, {'measure': 'Absolute Change in Fecal Elastase-1 (FE-1) Levels', 'timeFrame': 'From Parent Study 115B Baseline at Week 96'}, {'measure': 'Absolute Change in Immunoreactive Trypsinogen (IRT) Serum Levels', 'timeFrame': 'From Parent Study 115B Baseline at Week 96'}, {'measure': 'Number of Participants With Microbiology Culture Status (Positive or Negative)', 'timeFrame': 'Week 96', 'description': 'Following microbial tests were performed: Burkholderia, Methicillin Resistant Staphylococcus Aureus (MRSA), Methicillin Susceptible Staphylococcus Aureus (MSSA), Pseudomonas Aeruginosa Mucoid (P. Aeruginosa Mucoid), P. Aeruginosa Non-Mucoid, P. Aeruginosa Small Colony Variant and Stenotrophomonas Maltophilia.'}, {'measure': 'Absolute Change in Lung Clearance Index (LCI) 2.5', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.'}, {'measure': 'Absolute Change in Lung Clearance Index (LCI) 5.0', 'timeFrame': 'From Parent Study 115B Baseline at Week 96', 'description': 'LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '33965000', 'type': 'DERIVED', 'citation': 'Hoppe JE, Chilvers M, Ratjen F, McNamara JJ, Owen CA, Tian S, Zahigian R, Cornell AG, McColley SA. Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study. Lancet Respir Med. 2021 Sep;9(9):977-988. doi: 10.1016/S2213-2600(21)00069-2. Epub 2021 May 6.'}]}, 'descriptionModule': {'briefSummary': 'A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nSubjects entering the Treatment Cohort must meet the following criteria:\n\n* Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study VX15-809-115 Part B (Study 115B, NCT02797132)\n* Willing to remain on a stable CF medication regimen through the Safety Follow-up Visit\n\nSubjects entering the Observational Cohort must meet 1 of the following criteria:\n\n* Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study 115B, but do not want to enroll in the Treatment Cohort.\n* Received at least 4 weeks of LUM/IVA treatment and completed visits up to Week 24 and the Safety Follow-up Visit, if required, of Study 115B but are not taking LUM/IVA at the end of the Study 115B Treatment Period (i.e., Week 24) because of a drug interruption and either did not receive Vertex approval to enroll in the Treatment Cohort or do not want to enroll in the Treatment Cohort.\n* Permanently discontinued LUM/IVA in Study 115B after receiving at least 4 weeks of treatment and remained in the study from the time of treatment discontinuation through the Week 24 Visit and Safety Follow-up Visit, if required.\n\nExclusion Criteria (Treatment Cohort Only):\n\n* Prematurely discontinued LUM/IVA treatment in Study 115B.\n* History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering LUM/IVA to the subject\n* History of drug intolerance or other serious reactions to LUM/IVA in Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor.\n* Subjects with a history of allergy or hypersensitivity to LUM/IVA.\n* Liver function test (LFT) abnormality meeting criteria for LUM/IVA treatment interruption at the completion of Study 115B, for which no convincing alternative etiology is identified.\n* QTc value at the completion of Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor\n* History of poor compliance with LUM/IVA and/or procedures in Study 115B as deemed by the investigator.\n* Participation in an investigational drug trial (including studies investigating LUM and/or IVA) other than Study 115B.'}, 'identificationModule': {'nctId': 'NCT03125395', 'briefTitle': 'A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 3, Rollover Study to Evaluate the Safety of Long-term Treatment With Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation', 'orgStudyIdInfo': {'id': 'VX16-809-116'}, 'secondaryIdInfos': [{'id': '2019-003112-31', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'LUM/IVA', 'description': 'LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.', 'interventionNames': ['Drug: LUM/IVA']}], 'interventions': [{'name': 'LUM/IVA', 'type': 'DRUG', 'otherNames': ['VX-809/VX-770', 'lumacaftor/ivacaftor'], 'description': 'Participants received LUM/IVA every q12h.', 'armGroupLabels': ['LUM/IVA']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94305', '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 Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Riley Hospital for Children at Indiana University Health', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55404', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': "Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minn", 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'facility': "Children's Mercy Hospital", 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '14222', 'city': 'Buffalo', 'state': 'New York', 'country': 'United States', 'facility': "The Lung & Cystic Fibrosis Center at Women's & Children's Hospital of Buffalo", 'geoPoint': {'lat': 42.88645, 'lon': -78.87837}}, {'zip': '27514', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina Hospitals', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '44106', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': "University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital", 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '23507', 'city': 'Norfolk', 'state': 'Virginia', 'country': 'United States', 'facility': "Children's Hospital of the King's Daughters", 'geoPoint': {'lat': 36.84681, 'lon': -76.28522}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': 'V6H 3N1', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "British Columbia's Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'The Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'H4A 3J1', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': "McGill University Health Centre, Glen Site, Montreal Children's Hospital", 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}