Ignite Creation Date:
2025-12-25 @ 2:38 AM
Ignite Modification Date:
2026-03-03 @ 10:45 PM
Study NCT ID:
NCT01712334
Status:
COMPLETED
Last Update Posted:
2014-05-01
First Post:
2012-10-19
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis
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': 'C568813', 'term': 'dornase alfa'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'genentech@druginfo.com', 'phone': '800-821-8590', 'title': 'Medical Communications', 'organization': 'Hoffman-LaRoche'}, 'certainAgreement': {'otherDetails': "The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'description': 'Safety population included all randomized participants who received treatment.', 'eventGroups': [{'id': 'EG000', 'title': 'eRapid Nebulizer', 'description': 'Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.', 'otherNumAtRisk': 85, 'otherNumAffected': 0, 'seriousNumAtRisk': 85, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Jet Nebulizer', 'description': 'Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.', 'otherNumAtRisk': 85, 'otherNumAffected': 0, 'seriousNumAtRisk': 85, 'seriousNumAffected': 0}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'OG000'}, {'value': '85', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'eRapid Nebulizer', 'description': 'Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.'}, {'id': 'OG001', 'title': 'Jet Nebulizer', 'description': 'Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '98.1', 'spread': '22.1', 'groupId': 'OG000'}, {'value': '97.2', 'spread': '20.7', 'groupId': 'OG001'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': "Ratio (Fieller's theorem)", 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '90', 'paramValue': '100.9', 'ciLowerLimit': '99.5', 'ciUpperLimit': '102.3', 'nonInferiorityType': 'NON_INFERIORITY_OR_EQUIVALENCE', 'testedNonInferiority': True, 'nonInferiorityComment': 'The ratio of the mean percent predicted FEV1 at the end of the eRapid treatment to the mean percent predicted FEV1 at the end of the LC Plus jet nebulizer treatment. The two nebulizers were considered equivalent if the 90% CI was within 80%-125%.'}], 'paramType': 'MEAN', 'timeFrame': 'At the end of each 2-week treatment period', 'description': 'Spirometry was performed according to American Thoracic Society standards. FEV1 is the amount of air that is forced out of the lungs in one second and was measured at the end of each 2-week treatment period. The percent predicted FEV1 was calculated as: Percent predicted FEV1 =FEV1 (L) / Predicted FEV1 (L) ×100.', 'unitOfMeasure': 'percent predicted', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Modified Intent-to-Treat (mITT) population included all randomized participants with baseline and endpoint FEV1 values for both treatment periods.'}, {'type': 'PRIMARY', 'title': 'Safety: Number of Participants With Adverse Events During Each Treatment Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'OG000'}, {'value': '85', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'eRapid Nebulizer', 'description': 'Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.'}, {'id': 'OG001', 'title': 'Jet Nebulizer', 'description': 'Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}, {'value': '24', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '4 Weeks', 'description': 'An adverse event was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population included all randomized participants who received treatment.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'eRapid Nebulizer Then Jet Nebulizer', 'description': 'Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks in Treatment Period 1 then Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks in Treatment Period 2.\n\n.'}, {'id': 'FG001', 'title': 'Jet Nebulizer Then eRapid Nebulizer', 'description': 'Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks in Treatment Period 1 then Pulmozyme® inhaled once daily by the Pari eRapid nebulizer for 2 weeks in Treatment Period 2.'}], 'periods': [{'title': 'Treatment Period 1', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '44'}, {'groupId': 'FG001', 'numSubjects': '43'}]}, {'type': 'Received Treatment', 'achievements': [{'groupId': 'FG000', 'numSubjects': '44'}, {'groupId': 'FG001', 'numSubjects': '41'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '44'}, {'groupId': 'FG001', 'numSubjects': '41'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Randomized in Error', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}]}, {'title': 'Treatment Period 2', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '44'}, {'groupId': 'FG001', 'numSubjects': '41'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '44'}, {'groupId': 'FG001', 'numSubjects': '41'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': '99 patients were enrolled. 96 unique patients entered the run-in period including 3 patients who entered the run-in period twice.', 'preAssignmentDetails': 'Patients received dornase alfa (Pulmozyme®) by LC Plus nebulizer in the 2-week run-in period prior to randomization. A total of 86 unique patients were randomized in the study in 87 randomization events. Of the randomized patients, 85 patients completed the study in two treatment sequences.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '85', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'All Participants', 'description': 'All participants received dornase alfa (Pulmozyme) inhaled once daily by the Pari eRapid nebulizer or the Pari LC Plus jet nebulizer for 2 weeks in Treatment Period 1 then crossed over to use the other nebulizer in Treatment Period 2 for 2 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '13.6', 'spread': '6.92', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '43', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '42', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Baseline measures were based on the modified Intent-to-Treat (mITT) population that included all participants randomized with both baseline and endpoint Forced Expiratory Volume in 1 second (FEV1) values for both treatment periods.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 99}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-03', 'completionDateStruct': {'date': '2013-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-03-31', 'studyFirstSubmitDate': '2012-10-19', 'resultsFirstSubmitDate': '2014-03-27', 'studyFirstSubmitQcDate': '2012-10-19', 'lastUpdatePostDateStruct': {'date': '2014-05-01', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2014-03-31', 'studyFirstPostDateStruct': {'date': '2012-10-23', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-05-01', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)', 'timeFrame': 'At the end of each 2-week treatment period', 'description': 'Spirometry was performed according to American Thoracic Society standards. FEV1 is the amount of air that is forced out of the lungs in one second and was measured at the end of each 2-week treatment period. The percent predicted FEV1 was calculated as: Percent predicted FEV1 =FEV1 (L) / Predicted FEV1 (L) ×100.'}, {'measure': 'Safety: Number of Participants With Adverse Events During Each Treatment Period', 'timeFrame': '4 Weeks', 'description': 'An adverse event was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events.'}]}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '33735508', 'type': 'DERIVED', 'citation': 'Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.'}, {'pmid': '25921451', 'type': 'DERIVED', 'citation': 'Sawicki GS, Chou W, Raimundo K, Trzaskoma B, Konstan MW. Randomized trial of efficacy and safety of dornase alfa delivered by eRapid nebulizer in cystic fibrosis patients. J Cyst Fibros. 2015 Nov;14(6):777-83. doi: 10.1016/j.jcf.2015.04.003. Epub 2015 Apr 25.'}]}, 'descriptionModule': {'briefSummary': 'This phase IV, multicenter, randomized, open-label, two-period crossover study will evaluate the comparable efficacy and safety of Pulmozyme (dornase alfa) delivered by the eRapid nebulizer system in patients with cystic fibrosis. Patients who have been receiving Pulmozyme once daily chronically for at least 6 months will continue to receive Pulmozyme once daily for a run-in period of 2 weeks using the Pari LC Plus nebulizer. Patients will then be randomized to receive in a crossover design Pulmozyme once daily for two treatment periods of 2 weeks each using either the Pari LC Plus or the eRapid nebulizer. Anticipated time on study treatment is 6 weeks.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male and female patients, \\>/= 6 years of age\n* Confirmed diagnosis of cystic fibrosis (CF)\n* Receiving Pulmozyme once daily chronically for treatment of CF for at least 6 months prior to screening\n* Percent predicted FEV1 \\>/= 40% at screening based on the Wang (males \\< 18 years, females \\< 16 years) or Hankinson (males \\>/= 18 years, females \\>/= 16 years) standardized equations\n* Able to reproducibly perform spirometry testing and comply with study assessments\n\nExclusion Criteria:\n\n* An acute respiratory infection or pulmonary exacerbation within 4 weeks prior to randomization\n* Initiation of any new chronic therapy (e.g. inhaled corticosteroids, inhaled oral antibiotics, high-dose ibuprofen, hypertonic saline, ivacaftor) for respiratory disease within 4 weeks prior to randomization\n* Changes in chest physiotherapy schedule within 4 weeks prior to randomization\n* Hospitalization within 4 weeks prior to randomization\n* Planned hospitalization during the 6-week study\n* History of organ transplantation\n* Participation in an investigational drug or device study within 30 day prior to screening'}, 'identificationModule': {'nctId': 'NCT01712334', 'briefTitle': 'A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Genentech, Inc.'}, 'officialTitle': 'A Phase IV Multicenter, Randomized, Open Label, Two-Period, Crossover Study in Patients With Cystic Fibrosis to Evaluate the Comparable Efficacy and Safety of Pulmozyme Delivered by the eRapid Nebulizer System', 'orgStudyIdInfo': {'id': 'ML28249'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'eRapid Nebulizer', 'description': 'Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.', 'interventionNames': ['Drug: dornase alfa [Pulmozyme®]']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Jet Nebulizer', 'description': 'Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.', 'interventionNames': ['Drug: dornase alfa [Pulmozyme®]']}], 'interventions': [{'name': 'dornase alfa [Pulmozyme®]', 'type': 'DRUG', 'description': 'Inhaled once daily by Pari eRapid nebulizer.', 'armGroupLabels': ['eRapid Nebulizer']}, {'name': 'dornase alfa [Pulmozyme®]', 'type': 'DRUG', 'description': 'Inhaled once daily by Pari LC Plus jet nebulizer.', 'armGroupLabels': ['Jet Nebulizer']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85016', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '72202', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '4102', 'city': 'Portland', 'state': 'Maine', 'country': 'United States', 'geoPoint': {'lat': 43.65737, 'lon': -70.2589}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '03756', 'city': 'Lebanon', 'state': 'New Hampshire', 'country': 'United States', 'geoPoint': {'lat': 43.64229, 'lon': -72.25176}}, {'zip': '07740', 'city': 'Long Branch', 'state': 'New Jersey', 'country': 'United States', 'geoPoint': {'lat': 40.30428, 'lon': -73.99236}}, {'zip': '14618', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44308', 'city': 'Akron', 'state': 'Ohio', 'country': 'United States', 'geoPoint': {'lat': 41.08144, 'lon': -81.51901}}, {'zip': '44106', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '57104', 'city': 'Sioux Falls', 'state': 'South Dakota', 'country': 'United States', 'geoPoint': {'lat': 43.54369, 'lon': -96.72796}}, {'zip': '37232-9119', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}], 'overallOfficials': [{'name': 'Clinical Trials', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Genentech, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genentech, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}