Ignite Creation Date:
2025-12-24 @ 9:46 PM
Ignite Modification Date:
2026-01-26 @ 5:38 AM
Study NCT ID:
NCT02135432
Status:
COMPLETED
Last Update Posted:
2017-02-24
First Post:
2014-04-15
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
TOPIC Trial for COPD
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D029424', 'term': 'Pulmonary Disease, Chronic Obstructive'}], 'ancestors': [{'id': 'D008173', 'term': 'Lung Diseases, Obstructive'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C545203', 'term': 'ivacaftor'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'smrowe@uab.edu', 'phone': '205-975-6385', 'title': 'Steven M. Rowe', 'organization': 'University of Alabama at Birmingham'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'this pilot trial was under powered and potentially too brief to detect definitive changes in lung function'}}, 'adverseEventsModule': {'timeFrame': '1 year, 5 months', 'eventGroups': [{'id': 'EG000', 'title': 'Ivacaftor (VX-770)', 'description': 'twice a day administration of Ivacaftor: 150mg\n\nIvacaftor', 'otherNumAtRisk': 8, 'otherNumAffected': 7, 'seriousNumAtRisk': 8, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Placebo', 'description': 'matching placebo\n\nPlacebo', 'otherNumAtRisk': 4, 'otherNumAffected': 3, 'seriousNumAtRisk': 4, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'decreased/diminished breath sounds', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'COPD Exacerbation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Loose Stools', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'COPD Exacerbation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in COPD as Measured by the Sweat Analysis in Each Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ivacaftor (VX-770)', 'description': 'twice a day administration of Ivacaftor: 150mg\n\nIvacaftor'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'matching placebo\n\nPlacebo'}], 'classes': [{'categories': [{'measurements': [{'value': '-8.0', 'spread': '4.4', 'groupId': 'OG000'}, {'value': '2.0', 'spread': '1.5', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline to 2 weeks', 'description': 'sweat analysis is measured by performing a sweat test in each participant. The primary analysis will compare the within group change in sweat chloride before (day 1) and after (day 14) ivacaftor or placebo administration and will be used to test the null hypothesis of no change in sweat chloride using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.', 'unitOfMeasure': 'mmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': '8 patients were analyzed in the ivacaftor Arm because 8 patients were randomized to study drug and only 4 patients were randomized in the placebo arm because 4 patients received placebl'}, {'type': 'SECONDARY', 'title': 'Change in COPD as Measured by Nasal Potential Difference', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ivacaftor', 'description': 'patients randomized to ivacaftor twice daily'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'matching placebo\n\nPlacebo'}], 'classes': [{'categories': [{'measurements': [{'value': '-4.9', 'spread': '3.9', 'groupId': 'OG000'}, {'value': '1.0', 'spread': '6.4', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline to 2 weeks', 'description': 'Evaluate the efficacy of ivacaftor treatment in patients with COPD including measures of CFTR activity and clinical outcome as measured by change in nasal potential difference measurement in each group. These data will be used to test the null hypothesis of no change in nasal potential difference (ΔLow Chloride plus isoproterenol) using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.', 'unitOfMeasure': 'millivolts', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ivacaftor (VX-770)', 'description': 'twice a day administration of Ivacaftor: 150mg\n\nIvacaftor'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'matching placebo\n\nPlacebo'}], 'classes': [{'categories': [{'measurements': [{'value': '22', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'baseline to 2 weeks', 'description': 'Number of adverse events per subject in each the Ivacaftor subjects and placebo subjects', 'unitOfMeasure': 'adverse events', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ivacaftor (VX-770)', 'description': 'twice a day administration of Ivacaftor: 150mg\n\nIvacaftor'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'matching placebo\n\nPlacebo'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.3', 'spread': '5.2', 'groupId': 'OG000'}, {'value': '1.8', 'spread': '9.3', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'baseline to 2 weeks', 'description': 'Spirometry will be analyzed by ATS criteria, and the best of three reproducible efforts will be used to calculate FEV1 in comparison to Hankinson standards. The primary analysis will be the change in FEV1% from day 0 to day 14 within subject, and will be tested against the null hypothesis that no change occurs using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.', 'unitOfMeasure': 'percentage of FEV1', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Pharmacokinetics as Described by AUC12 of Subjects Receiving Ivacaftor', 'timeFrame': 'baseline to 2 weeks', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ivacaftor (VX-770)', 'description': 'twice a day administration of Ivacaftor: 150mg\n\nIvacaftor'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'matching placebo\n\nPlacebo'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '4'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '4'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Ivacaftor (VX-770)', 'description': 'twice a day administration of Ivacaftor: 150mg\n\nIvacaftor'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'matching placebo\n\nPlacebo'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '55', 'groupId': 'BG000', 'lowerLimit': '49', 'upperLimit': '65'}, {'value': '62', 'groupId': 'BG001', 'lowerLimit': '60', 'upperLimit': '65'}, {'value': '55', 'groupId': 'BG002', 'lowerLimit': '49', 'upperLimit': '65'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Age, Customized', 'classes': [{'title': '<=18 years', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Between 18 and 65 years', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}]}, {'title': '>=65 years', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Gender', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-01', 'completionDateStruct': {'date': '2015-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-01-05', 'studyFirstSubmitDate': '2014-04-15', 'resultsFirstSubmitDate': '2016-05-25', 'studyFirstSubmitQcDate': '2014-05-08', 'lastUpdatePostDateStruct': {'date': '2017-02-24', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-01-05', 'studyFirstPostDateStruct': {'date': '2014-05-12', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-02-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2015-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Pharmacokinetics as Described by AUC12 of Subjects Receiving Ivacaftor', 'timeFrame': 'baseline to 2 weeks'}], 'primaryOutcomes': [{'measure': 'Change in COPD as Measured by the Sweat Analysis in Each Group', 'timeFrame': 'baseline to 2 weeks', 'description': 'sweat analysis is measured by performing a sweat test in each participant. The primary analysis will compare the within group change in sweat chloride before (day 1) and after (day 14) ivacaftor or placebo administration and will be used to test the null hypothesis of no change in sweat chloride using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.'}], 'secondaryOutcomes': [{'measure': 'Change in COPD as Measured by Nasal Potential Difference', 'timeFrame': 'baseline to 2 weeks', 'description': 'Evaluate the efficacy of ivacaftor treatment in patients with COPD including measures of CFTR activity and clinical outcome as measured by change in nasal potential difference measurement in each group. These data will be used to test the null hypothesis of no change in nasal potential difference (ΔLow Chloride plus isoproterenol) using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.'}, {'measure': 'Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.', 'timeFrame': 'baseline to 2 weeks', 'description': 'Number of adverse events per subject in each the Ivacaftor subjects and placebo subjects'}, {'measure': 'Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group', 'timeFrame': 'baseline to 2 weeks', 'description': 'Spirometry will be analyzed by ATS criteria, and the best of three reproducible efforts will be used to calculate FEV1 in comparison to Hankinson standards. The primary analysis will be the change in FEV1% from day 0 to day 14 within subject, and will be tested against the null hypothesis that no change occurs using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Chronic Obstructive pulmonary disease, Ivacaftor'], 'conditions': ['Chronic Obstructive Pulmonary Disease']}, 'descriptionModule': {'briefSummary': 'The study is a randomized, double-blind, placebo-controlled, multiple-dose, pilot study of orally-administered ivacaftor in subjects with chronic obstructive pulmonary disease. Subjects will be administered the study drug ivacaftor 150 mg (or placebo) twice daily (BID).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '40 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Male or Female age 40-65\n* A clinical diagnosis of COPD as defined by GOLD\n* At Least a 10 pack year smoking history\n* Exhibit symptoms of chronic bronchitis defined by MRC\n* FEV1% predicted ≥ 35% and ≤70% Post Bronchodilator\n* Clinically stable in the last 4 weeks with no evidence of COPD exacerbation\n* Weight of 40 kg-120 kg\n* Willingness to use at least one form of acceptable birth control including abstinence, condom with spermicide, or hormonal contraceptives\n* Willing to monitor blood glucose if known history of diabetes mellitus requiring insulin or medical therapy\n\nExclusion Criteria:\n\n* Current Diagnosis of Asthma\n* Daytime use of Oxygen Therapy\n* Documented history of drug abuse within the last year\n* Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease within 28 days before receiving the first dose of study drug.\n* Cirrhosis or elevated liver transaminases \\> 3X ULN\n* GFR \\< 50 estimated by Cockcroft-Gault\n* Any illness of abnormal lab finding that, in the opinion of the investigator or the subject's general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject.\n* Pregnant or Breastfeeding\n* Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit/grapefruit juice.\n* Uncontrolled Diabetes\n* Excluded medications and foods include the drugs and foods provided in the appendix document.\n* Clinically significant arrhythmias or conduction abnormalities that in the opinion of the investigator affect patient safety have been added as exclusion criteria and criteria for withdrawal.\n\nPatients who have not been stable or have been hospitalized in the past 3 months with any clinically significant cardiac conditions.\n\nSubjects with history of cancer (current or past, unless remote (\\>5years))except for localized non-melanomatous skin cancers History of Stroke/CVA History of myocardial infarction/acute coronary syndrome Cardiac Failure NYHC grade III-IV Diabetes Type I Uncontrolled Hypertension Primary or secondary pulmonary hypertension\n\n\\-"}, 'identificationModule': {'nctId': 'NCT02135432', 'briefTitle': 'TOPIC Trial for COPD', 'organization': {'class': 'OTHER', 'fullName': 'University of Alabama at Birmingham'}, 'officialTitle': 'A Randomized, Double-Blind, Placebo Controlled Pilot Study to Determine the Safety and Efficacy of Ivacaftor (VX-770) for the Treatment of Chronic Obstructive Pulmonary Disease (The TOPIC Trial)', 'orgStudyIdInfo': {'id': 'F140319002'}, 'secondaryIdInfos': [{'id': 'TOPIC Trial', 'type': 'OTHER', 'domain': 'UAB'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ivacaftor (VX-770)', 'description': 'twice a day administration of Ivacaftor: 150mg', 'interventionNames': ['Drug: Ivacaftor']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'matching placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Ivacaftor', 'type': 'DRUG', 'otherNames': ['Kalydeco'], 'armGroupLabels': ['Ivacaftor (VX-770)']}, {'name': 'Placebo', 'type': 'DRUG', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Alabama at Birmingham', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD', 'investigatorFullName': 'Dr. Steven M Rowe', 'investigatorAffiliation': 'University of Alabama at Birmingham'}}}}