Ignite Creation Date:
2025-12-25 @ 1:27 AM
Ignite Modification Date:
2025-12-25 @ 11:38 PM
Study NCT ID:
NCT03445793
Status:
COMPLETED
Last Update Posted:
2024-01-23
First Post:
2018-02-05
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)
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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'taylorcousarj@njhealth.org', 'phone': '3032702764', 'title': 'Jennifer Taylor-Cousar, Principal Investigator', 'organization': 'National Jewish Health'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'The limitation is the small sample size. Less patients with cystic fibrosis transitioned from lumacaftor/ivacaftor to tezacaftor/ivacaftor than expected. The enrolled number of patients left the study underpowered to detect differences.'}}, 'adverseEventsModule': {'timeFrame': '6 months', 'eventGroups': [{'id': 'EG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 2, 'seriousNumAtRisk': 5, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Increased cough after stopping Tezacaftor/Ivacaftor', 'notes': 'Subject stopped treatment with Tezacaftor/Ivacaftor after transitioning from Lumacaftor/Ivacaftor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Exacerbation of Chronic Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Hospitalization due to pulmonary exacerbation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 16, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Sweat Chloride Concentration in Millimoles/Liter From Baseline at 6 Months Pre-specified to be Reported', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'measurements': [{'value': '10.2', 'groupId': 'OG000', 'lowerLimit': '-12.1', 'upperLimit': '32.5'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to 6 months', 'description': 'Sweat chloride is a measure of cystic fibrosis transmembrane conductance regulator function. The calculations represent the average change from baseline to the average change at 6 months.', 'unitOfMeasure': 'mmol/L', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Rationale for Transition Per Physician Questionnaire', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'title': 'Side Effects/Intolerance of Orkambi', 'measurements': [{'value': '4', 'groupId': 'OG000'}]}, {'title': 'No benefit on Orkambi', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 day (the questionnaire is done once at visit 1)', 'description': "Questionnaire to determine the treating physician's reason for transition to tezacaftor/ivacaftor from lumacaftor/ivacaftor", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Rationale for Transition Per Subject Questionnaire', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'title': '"Orkambi is no longer working for me"', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': 'No benefit on Orkambi', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': 'Side effects/Intolerance on Orkambi', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': 'Personal desire to try new therapy', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 day (the questionnaire is done once at visit 1)', 'description': "Questionnaire to determine the subject's reason for transition to tezacaftor/ivacaftor from lumacaftor/ivacaftor", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Pulmonary Exacerbations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.2', 'spread': '3.96', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'One year prior to study entry (time of consent) and during study participation', 'description': 'Number of pulmonary exacerbations requiring oral or IV antibiotics', 'unitOfMeasure': 'Pulmonary Exacerbations', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Percent Predicted (ppFEV1) Value From Baseline at 6 Months Pre-specified to be Reported', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.0', 'groupId': 'OG000', 'lowerLimit': '-5.93', 'upperLimit': '1.93'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to 6 months', 'description': 'Pulmonary function by spirometry, percent predicted forced expiratory volume in 1 second. The calculations represent the average change from baseline to the average change at 6 months.', 'unitOfMeasure': 'percent predicted (ppFEV1)', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline at 6 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'measurements': [{'value': '-11.3', 'groupId': 'OG000', 'lowerLimit': '-25.9', 'upperLimit': '3.2'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to 6 months', 'description': 'CF-related quality of life measure is a validated, CF specific, patient reported outcome (PRO). This portion of the PRO is specific to respiratory symptoms. The scaled score for each domain ranges from 0 (worst condition) to 100 (best condition), with higher scores indicating better health in respiratory domain.', 'unitOfMeasure': 'score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Weight in Kilograms From Baseline at 6 Months Pre-specified to be Reported', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.0', 'spread': '2.68', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to 6 months', 'description': 'Weight is a reflection of nutrition status in CF and is expected to improve with CFTR modulation. The calculations represent the average change from baseline to the average change at 6 months.', 'unitOfMeasure': 'kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in BMI in kg/m^2 From Baseline at 6 Months Pre-specified to be Reported', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.67', 'groupId': 'OG000', 'lowerLimit': '-1.83', 'upperLimit': '0.48'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to 6 months', 'description': 'BMI is a reflection of nutrition status in CF and is expected to improve with CFTR modulation. The calculations represent the average change from baseline to the average change at 6 months.', 'unitOfMeasure': 'kg/m^2', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of People With Undetectable or Normal Fecal Elastase Measurements at 6 Months Pre-specified to be Reported', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'title': 'Undetectable <15', 'measurements': [{'value': '5', 'groupId': 'OG000'}]}, {'title': 'Normal Result', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 months', 'description': 'Fecal elastase in micrograms/gram. Fecal elastase is a measure of pancreatic function: Less than 100 mcg/g indicates severe exocrine pancreatic insufficiency, 100-200 mcg/g indicates moderate to mild insufficiency, greater than 200 indicates normal pancreatic function. The count represents the number of participants with either an undetectable fecal elastase level or normal level at 6 months, indicating change in pancreatic function at 6 months.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Gastrointestinal Symptom Tracker Score From Baseline to 6 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.6', 'spread': '8.20', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to 6 months', 'description': 'The Exocrine Pancreatic Insufficiency (EPI) GI Symptom Tracker is meant to measure the impact treatment is having on GI symptoms, perceived by person completing the tracker. Participants answer 24 symptom questions on a scale from almost always to never (scale 4 to 1). Higher scores reflect more challenges/problems related to GI symptoms. For purposes of this outcome measure, subscales were combined to compute a total score (minimum score 24, maximum score 96). The difference of total scores from baseline at 6 months were calculated to measure average overall change in GI symptoms.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Single center recruitment from March 2018 until November 2019.', 'preAssignmentDetails': 'Participant must complete baseline visit while on lumacaftor/ivacaftor prior to transition to tezacaftor/ivacaftor.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Observational', 'description': 'Participants who have cystic fibrosis and are clinically stable at time of transition from Lumacaftor/Ivacaftor to tezacaftor/ivacaftor as a continuous treatment as determined by their clinical physician.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '28.4', 'spread': '1.14', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'Age at time of consent', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '3', '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': '5', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Cystic Fibrosis Mutation Type', 'classes': [{'categories': [{'title': 'Homozygous for F508del', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Heterozygous for F508del', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'BMI', 'classes': [{'categories': [{'measurements': [{'value': '22.3', 'spread': '1.50', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg/m^2', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-03-26', 'size': 985204, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2020-04-14T12:50', 'hasProtocol': True}, {'date': '2019-02-19', 'size': 439304, 'label': 'Informed Consent Form', 'hasIcf': True, 'hasSap': False, 'filename': 'ICF_001.pdf', 'typeAbbrev': 'ICF', 'uploadDate': '2020-04-14T12:52', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-03-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-01', 'completionDateStruct': {'date': '2019-11-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-01-02', 'studyFirstSubmitDate': '2018-02-05', 'resultsFirstSubmitDate': '2020-06-05', 'studyFirstSubmitQcDate': '2018-02-22', 'lastUpdatePostDateStruct': {'date': '2024-01-23', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-09-03', 'studyFirstPostDateStruct': {'date': '2018-02-26', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2021-10-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-11-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Sweat Chloride Concentration in Millimoles/Liter From Baseline at 6 Months Pre-specified to be Reported', 'timeFrame': 'Baseline to 6 months', 'description': 'Sweat chloride is a measure of cystic fibrosis transmembrane conductance regulator function. The calculations represent the average change from baseline to the average change at 6 months.'}], 'secondaryOutcomes': [{'measure': 'Rationale for Transition Per Physician Questionnaire', 'timeFrame': '1 day (the questionnaire is done once at visit 1)', 'description': "Questionnaire to determine the treating physician's reason for transition to tezacaftor/ivacaftor from lumacaftor/ivacaftor"}, {'measure': 'Rationale for Transition Per Subject Questionnaire', 'timeFrame': '1 day (the questionnaire is done once at visit 1)', 'description': "Questionnaire to determine the subject's reason for transition to tezacaftor/ivacaftor from lumacaftor/ivacaftor"}, {'measure': 'Pulmonary Exacerbations', 'timeFrame': 'One year prior to study entry (time of consent) and during study participation', 'description': 'Number of pulmonary exacerbations requiring oral or IV antibiotics'}, {'measure': 'Change in Percent Predicted (ppFEV1) Value From Baseline at 6 Months Pre-specified to be Reported', 'timeFrame': 'Baseline to 6 months', 'description': 'Pulmonary function by spirometry, percent predicted forced expiratory volume in 1 second. The calculations represent the average change from baseline to the average change at 6 months.'}, {'measure': 'Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline at 6 Months', 'timeFrame': 'Baseline to 6 months', 'description': 'CF-related quality of life measure is a validated, CF specific, patient reported outcome (PRO). This portion of the PRO is specific to respiratory symptoms. The scaled score for each domain ranges from 0 (worst condition) to 100 (best condition), with higher scores indicating better health in respiratory domain.'}, {'measure': 'Change in Weight in Kilograms From Baseline at 6 Months Pre-specified to be Reported', 'timeFrame': 'Baseline to 6 months', 'description': 'Weight is a reflection of nutrition status in CF and is expected to improve with CFTR modulation. The calculations represent the average change from baseline to the average change at 6 months.'}, {'measure': 'Change in BMI in kg/m^2 From Baseline at 6 Months Pre-specified to be Reported', 'timeFrame': 'Baseline to 6 months', 'description': 'BMI is a reflection of nutrition status in CF and is expected to improve with CFTR modulation. The calculations represent the average change from baseline to the average change at 6 months.'}, {'measure': 'Number of People With Undetectable or Normal Fecal Elastase Measurements at 6 Months Pre-specified to be Reported', 'timeFrame': '6 months', 'description': 'Fecal elastase in micrograms/gram. Fecal elastase is a measure of pancreatic function: Less than 100 mcg/g indicates severe exocrine pancreatic insufficiency, 100-200 mcg/g indicates moderate to mild insufficiency, greater than 200 indicates normal pancreatic function. The count represents the number of participants with either an undetectable fecal elastase level or normal level at 6 months, indicating change in pancreatic function at 6 months.'}, {'measure': 'Change in Gastrointestinal Symptom Tracker Score From Baseline to 6 Months', 'timeFrame': 'Baseline to 6 months', 'description': 'The Exocrine Pancreatic Insufficiency (EPI) GI Symptom Tracker is meant to measure the impact treatment is having on GI symptoms, perceived by person completing the tracker. Participants answer 24 symptom questions on a scale from almost always to never (scale 4 to 1). Higher scores reflect more challenges/problems related to GI symptoms. For purposes of this outcome measure, subscales were combined to compute a total score (minimum score 24, maximum score 96). The difference of total scores from baseline at 6 months were calculated to measure average overall change in GI symptoms.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['DeltaF508deletion (F508del) mutation in 2 copies'], 'conditions': ['Cystic Fibrosis (CF)']}, 'descriptionModule': {'briefSummary': 'This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva. Clinical and laboratory measurements will be measured at baseline, 1 month, 3 months, and 6 months after initiation of tez/iva. Change from baseline at 6 months pre-specified will be reported. The length of study participation will be approximately 6 months.', 'detailedDescription': 'While cystic fibrosis (CF) therapeutic development previously targeted the signs and symptoms of the disease, in the last 5 years, two drugs that treat the basic defect in CF have been approved, ivacaftor (iva) and lumacaftor/ivacaftor (lum/iva). This class of drugs, deemed cystic fibrosis transmembrane conductance regulator (CFTR) modulators, variably improve CFTR function as measured by pilocarpine iontophoresis and sweat collection, and clinical outcomes including lung function, body mass index (BMI), rate of exacerbations and patient reported quality of life.\n\nIn patients with the G551D mutation who received iva, there was a marked decrease in sweat chloride and marked improvement in lung function as measured by absolute change from baseline of percent predicted expiratory volume in 1 second (ppFEV1). The clinical outcomes assessed in the phase III studies of lum/iva and tez/iva were similar, and included lung function, rate of pulmonary exacerbations, BMI, and CFQ-R scores. While the correlation between improvement in sweat chloride and lung function is poor, and a minimum threshold for change in sweat chloride that correlates with clinical outcomes has yet to be defined, it has also not been determined if an increase in sweat chloride caused by a transition from one drug to another would adversely impact clinical outcomes. Outcomes between the phase III studies of lum/iva and tez/iva were similar, tez/iva has three advantages that are likely to make it more appealing to patients and providers than lum/iva including positive clinical efficacy data, fewer drug-drug interactions, and an improved tolerance profile.\n\nFollowing tez/iva approval, a rapid uptake of tez/iva for patients homozygous for F508del CFTR is expected; as a result of the transition from lum/iva to tez/iva, sweat chloride will increase possibly resulting in an adverse impact on clinical outcomes. This study aims to determine the rationale for patient transition from lum/iva to tez/iva, in addition to evaluate the impact of transition on CFTR function, pulmonary health, gastrointestinal health, and general health. While it is possible that there will be no change in sweat chloride or small changes that are without clinical significance, systematic collection of data as patients transition from lum/iva to tez/iva would permit rapid identification of any safety issues. Because the U.S. always leads the way with approval and reimbursement of new therapeutics, our experience with this transition will help guide its conduct for physicians and patients in the rest of the world.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'Cystic fibrosis with 2 copies of F508del mutation.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed diagnosis of CF\n* Male or female subjects greater than or equal to 12 years of age\n* Ability to reproducibly perform spirometry testing\n* Physician decision to treat with tezacaftor/ivacaftor (Smydeko)\n* Ability to understand and sign a written informed consent or assent and comply with the requirements of the study\n* Continuous use of orkambi for at least 1 month prior to visit 1\n\nExclusion Criteria:\n\n* History of hypersensitivity to tezacaftor and/or ivacaftor\n* Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data\n* Any acute lower respiratory symptoms treated with oral, inhaled or intravenous antibiotics (IV) or systemic corticosteroids within the 2 weeks prior to Visit 1\n* Major or traumatic surgery within 12 weeks prior to Visit 1\n* For women of child-bearing potential: a positive pregnancy test at Visit 1\n* Unable or unwilling to fast (including no enteric tube feedings) for at least 6 hours prior each visit\n* Initiation of any new chronic therapy within 4 weeks prior to Visit 1\n* Use of an investigational agent within 28 days prior to Visit 1\n* Use of chronic oral corticosteroids within 28 days prior to Visit 1\n* Treatment for nontuberculous mycobacterial (NTM) infection, consisting of greater than or equal to two antibiotics (oral, IV, and/or inhaled) within 28 days prior to Visit 1\n* History of lung or liver transplantation, or listing for organ transplantation'}, 'identificationModule': {'nctId': 'NCT03445793', 'briefTitle': ': TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)', 'organization': {'class': 'OTHER', 'fullName': 'National Jewish Health'}, 'officialTitle': ': TRANSITION: An Observational Study of the Effects on Sweat Chloride and Clinical Outcomes of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)', 'orgStudyIdInfo': {'id': 'HS-3145'}}, 'contactsLocationsModule': {'locations': [{'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'National Jewish Heatlh', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}], 'overallOfficials': [{'name': 'Jennifer Taylor-Cousar, MD, MSCS', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'National Jewish Health'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Jewish Health', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD, Principal Investigator, Co-Director/CF TDN Director, Adult CF Program', 'investigatorFullName': 'Jennifer Taylor-Cousar', 'investigatorAffiliation': 'National Jewish Health'}}}}