Ignite Creation Date:
2025-12-26 @ 11:13 PM
Ignite Modification Date:
2025-12-26 @ 11:13 PM
Study NCT ID:
NCT00630812
Status:
COMPLETED
Last Update Posted:
2020-10-09
First Post:
2008-02-27
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Long Term Administration of Inhaled Mannitol in 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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Brett.Charlton@pharmaxis.com.au', 'phone': '02 94547210', 'title': 'Brett Charlton', 'organization': 'Pharmaxis'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': '26 weeks', 'description': 'Safety information reported is for the 26 week double blind period only', 'eventGroups': [{'id': 'EG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks', 'otherNumAtRisk': 184, 'deathsNumAtRisk': 184, 'otherNumAffected': 165, 'seriousNumAtRisk': 184, 'deathsNumAffected': 0, 'seriousNumAffected': 31}, {'id': 'EG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase', 'otherNumAtRisk': 121, 'deathsNumAtRisk': 121, 'otherNumAffected': 106, 'seriousNumAtRisk': 121, 'deathsNumAffected': 1, 'seriousNumAffected': 30}], 'otherEvents': [{'term': 'Condition Aggravated', 'notes': 'Pulmonary Exacerbations were coded to condition aggravated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 57}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 41}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 28}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 16}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 26}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 22}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pharyngolaryngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 19}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 13}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 13}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 11}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}], 'seriousEvents': [{'term': 'Condition Aggravated', 'notes': 'pulmonary exacerbations were coded to condition aggravated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 27}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 20}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Asthmatic Crisis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pleuritic Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumothorax', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Bronchopneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Acute tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pneumonia bacterial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pyelonephritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pancreatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Distal intestinal obstruction syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Intestinal obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Pancreatis acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Cholecystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}, {'term': 'Diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 184, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 121, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (11.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change in Absolute FEV1 From Baseline Over 26 Weeks', 'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'OG000'}, {'value': '121', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'measurements': [{'value': '106.53', 'groupId': 'OG000', 'lowerLimit': '62.43', 'upperLimit': '150.62'}, {'value': '52.38', 'groupId': 'OG001', 'lowerLimit': '2.09', 'upperLimit': '102.68'}]}]}], 'analyses': [{'pValue': '0.059', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '54.14', 'ciLowerLimit': '-1.97', 'ciUpperLimit': '110.26', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.041', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Odds Ratio (OR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.69', 'ciLowerLimit': '1.02', 'ciUpperLimit': '2.80', 'pValueComment': 'Response defined as change of \\>=100mL at week 26', 'statisticalMethod': 'Regression, Logistic', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': '45.7% response on Mannitol 400mg, 35.5% on Control'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '26 weeks', 'description': 'Change from baseline in forced expiratory volume at one second (FEV1) averaged over 26 weeks (measured at 6,14 and 26 weeks) The mean absolute change from baseline FEV1 (mL) over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach.Least square means presented are for the average change over the 6, 14, and 26 week visits.', 'unitOfMeasure': 'mL', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'mITT (modified intent to treat) - patients randomised and treated with at least one dose of study medication)'}, {'type': 'SECONDARY', 'title': 'Change in FEV1 From Baseline Over 26 Weeks - Dornase Users', 'denoms': [{'units': 'Participants', 'counts': [{'value': '137', 'groupId': 'OG000'}, {'value': '92', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'measurements': [{'value': '78.60', 'groupId': 'OG000', 'lowerLimit': '27.64', 'upperLimit': '129.56'}, {'value': '35.11', 'groupId': 'OG001', 'lowerLimit': '-20.99', 'upperLimit': '91.21'}]}]}], 'analyses': [{'pValue': '0.177', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '43.49', 'ciLowerLimit': '-19.8', 'ciUpperLimit': '106.78', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '26 weeks', 'description': 'In the subset of dornase users, the mean absolute change from baseline FEV1 (mL) averaged over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits.\n\nChange from baseline over 26 weeks (measured at 6,14, 26 weeks) in subset of dornase users', 'unitOfMeasure': 'mL', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Note these are subset of dornase users - effect in dornase users was estimated using a model fitted using data from all patients'}, {'type': 'SECONDARY', 'title': 'Rate of Protocol Defined Pulmonary Exacerbations (PDPE)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'OG000'}, {'value': '121', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'title': 'With 1 PDPE', 'measurements': [{'value': '21', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}, {'title': 'With 2 PDPE', 'measurements': [{'value': '6', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}, {'title': 'With 3 PDPE', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}, {'title': 'No PDPEs', 'measurements': [{'value': '156', 'groupId': 'OG000'}, {'value': '98', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.520', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Rate ratio', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.85', 'ciLowerLimit': '0.51', 'ciUpperLimit': '1.41', 'statisticalMethod': 'Poisson regression', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Offset of the natural logarithm of follow-up time'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '26 weeks', 'description': 'Exacerbations treated with IV antibiotics and with at least 4 signs and symptoms according to Fuchs criteria (1994). Summary table presents the number with 0, 1,2 and 3 PDPEs during the 26 week treatment period.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'mITT (randomised and treated)'}, {'type': 'SECONDARY', 'title': 'Hospitalisations Associated With Protocol Defined Pulmonary Exacerbations (PDPEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'OG000'}, {'value': '121', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'title': '0 hospitalisations', 'measurements': [{'value': '162', 'groupId': 'OG000'}, {'value': '102', 'groupId': 'OG001'}]}, {'title': '1 hospitalisation', 'measurements': [{'value': '18', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}]}, {'title': '2 hospitalisations', 'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}, {'title': '3 hospitalisations', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.328', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Rate ratio', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.75', 'ciLowerLimit': '0.42', 'ciUpperLimit': '1.33', 'statisticalMethod': 'Poisson regression', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Offset of the natural logarithm of follow-up time'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '26 weeks', 'description': 'The number of hospitalisations is summarised and then the rate per person is analysed.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Antibiotic Use Associated With PDPEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'OG000'}, {'value': '121', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'title': '0 courses', 'measurements': [{'value': '156', 'groupId': 'OG000'}, {'value': '98', 'groupId': 'OG001'}]}, {'title': '1 course', 'measurements': [{'value': '22', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}, {'title': '2 courses', 'measurements': [{'value': '5', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}, {'title': '3 courses', 'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.368', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Rate ratio', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.89', 'ciLowerLimit': '0.69', 'ciUpperLimit': '1.15', 'statisticalMethod': 'Poisson regression', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '26 weeks', 'description': 'Number of courses per person in the 26 week period is summarised and then the rate per person analysed.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Absolute Change in FEV1 Percent Predicted at 26 Weeks', 'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'OG000'}, {'value': '121', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'measurements': [{'value': '3.14', 'groupId': 'OG000', 'lowerLimit': '1.49', 'upperLimit': '4.78'}, {'value': '0.72', 'groupId': 'OG001', 'lowerLimit': '-1.18', 'upperLimit': '2.62'}]}]}], 'analyses': [{'pValue': '0.024', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.42', 'ciLowerLimit': '0.33', 'ciUpperLimit': '4.51', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '26 weeks', 'description': 'Change from baseline at 26 weeks in FEV1 percent predicted with BOCF for those with missing values at week 26', 'unitOfMeasure': '% of predicted', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Baseline Observation carried forward (BOCF) implemented for those with missing values at visit 4'}, {'type': 'SECONDARY', 'title': 'Change in FVC (mL) Across 26 Weeks', 'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'OG000'}, {'value': '121', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'measurements': [{'value': '136.33', 'groupId': 'OG000', 'lowerLimit': '88.54', 'upperLimit': '184.11'}, {'value': '64.98', 'groupId': 'OG001', 'lowerLimit': '10.58', 'upperLimit': '119.37'}]}]}], 'analyses': [{'pValue': '0.022', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '71.35', 'ciLowerLimit': '10.57', 'ciUpperLimit': '132.13', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '26 weeks', 'description': 'Change from baseline in forced vital capacity (FVC) across 26 weeks (measured at 6,14 and 26 weeks)', 'unitOfMeasure': 'mL', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline FEF25-75 (mL/s) Over 26 Weeks', 'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'OG000'}, {'value': '121', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'measurements': [{'value': '84.65', 'groupId': 'OG000', 'lowerLimit': '6.66', 'upperLimit': '162.63'}, {'value': '50.31', 'groupId': 'OG001', 'lowerLimit': '-38.24', 'upperLimit': '138.86'}]}]}], 'analyses': [{'pValue': '0.49', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '34.34', 'ciLowerLimit': '-63.47', 'ciUpperLimit': '132.14', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '26 weeks', 'description': 'Change from baseline in forced expiratory flow at 25-75% of forced vital capacity (FEF25-75) (mL/s) averaged over 26 weeks (measured at 6,14 and 26 weeks) The mean absolute change from baseline over 26 weeks (measured at week 6, 14 and 26) was compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits.', 'unitOfMeasure': 'mL/s', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Sputum Weight at Baseline in Response to First Dose of Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '180', 'groupId': 'OG000'}, {'value': '114', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'OG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'classes': [{'categories': [{'measurements': [{'value': '4.9', 'spread': '6.18', 'groupId': 'OG000'}, {'value': '3.5', 'spread': '4.40', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.042', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'up to 30 mins after first dose of trial treatment', 'description': 'Sputum was collected during and for 30 minutes following the administration of the first dose of study treatment.', 'unitOfMeasure': 'g', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients randomised and treated who have sputum weight results available.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg twice a day (BD) for 26 + 26 weeks'}, {'id': 'FG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}], 'periods': [{'title': 'Randomisation to First Dose', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '192'}, {'groupId': 'FG001', 'numSubjects': '126'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '184'}, {'groupId': 'FG001', 'numSubjects': '121'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '5'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}, {'title': 'Double Blind Treatment Period', 'milestones': [{'type': 'STARTED', 'comment': 'Randomised and received first dose (mITT population). Patients not starting treatment are excluded', 'achievements': [{'groupId': 'FG000', 'numSubjects': '184'}, {'groupId': 'FG001', 'numSubjects': '121'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '153'}, {'groupId': 'FG001', 'numSubjects': '107'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '31'}, {'groupId': 'FG001', 'numSubjects': '14'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '5'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '7'}]}, {'type': 'Wanted to take drug in non-protocol way', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Non-compliance', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'Patients underwent a mannitol tolerance test (MTT) to gauge tolerance to a test dose of mannitol. 318 patients were then randomised - only 305 of the randomised patients went on to receive a dose of study medication. Patients who did not start medication (13) were not included in the modified ITT population (mITT)'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '184', 'groupId': 'BG000'}, {'value': '121', 'groupId': 'BG001'}, {'value': '305', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Mannitol 400mg', 'description': 'active treatment\n\ninhaled mannitol: 400 mg BD for 26 + 26 weeks'}, {'id': 'BG001', 'title': 'Control', 'description': 'Control (40mg inhaled mannitol) : BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '91', 'groupId': 'BG000'}, {'value': '63', 'groupId': 'BG001'}, {'value': '154', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '93', 'groupId': 'BG000'}, {'value': '58', 'groupId': 'BG001'}, {'value': '151', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '90', 'groupId': 'BG000'}, {'value': '58', 'groupId': 'BG001'}, {'value': '148', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '94', 'groupId': 'BG000'}, {'value': '63', 'groupId': 'BG001'}, {'value': '157', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '182', 'groupId': 'BG000'}, {'value': '119', 'groupId': 'BG001'}, {'value': '301', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Canada', 'categories': [{'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}]}, {'title': 'Argentina', 'categories': [{'measurements': [{'value': '48', 'groupId': 'BG000'}, {'value': '32', 'groupId': 'BG001'}, {'value': '80', 'groupId': 'BG002'}]}]}, {'title': 'Netherlands', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}, {'title': 'Belgium', 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '85', 'groupId': 'BG000'}, {'value': '54', 'groupId': 'BG001'}, {'value': '139', 'groupId': 'BG002'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Hospitalisations in year prior to study participation', 'classes': [{'categories': [{'title': '0 hospitalisations', 'measurements': [{'value': '114', 'groupId': 'BG000'}, {'value': '76', 'groupId': 'BG001'}, {'value': '190', 'groupId': 'BG002'}]}, {'title': '1 hospitalisation', 'measurements': [{'value': '41', 'groupId': 'BG000'}, {'value': '30', 'groupId': 'BG001'}, {'value': '71', 'groupId': 'BG002'}]}, {'title': '2 hospitalisations', 'measurements': [{'value': '21', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}]}, {'title': '3 hospitalisations', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}, {'title': '>3 hospitalisations', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Pulmonary exacerbations in year prior to study participation', 'classes': [{'categories': [{'title': '0 exacerbations', 'measurements': [{'value': '104', 'groupId': 'BG000'}, {'value': '74', 'groupId': 'BG001'}, {'value': '178', 'groupId': 'BG002'}]}, {'title': '1 exacerbation', 'measurements': [{'value': '45', 'groupId': 'BG000'}, {'value': '28', 'groupId': 'BG001'}, {'value': '73', 'groupId': 'BG002'}]}, {'title': '2 exacerbations', 'measurements': [{'value': '26', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '40', 'groupId': 'BG002'}]}, {'title': '3 exacerbations', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}, {'title': '>3 exacerbations', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'CF mutation', 'classes': [{'categories': [{'title': 'Both deltaF508', 'measurements': [{'value': '77', 'groupId': 'BG000'}, {'value': '45', 'groupId': 'BG001'}, {'value': '122', 'groupId': 'BG002'}]}, {'title': 'One deltaF508', 'measurements': [{'value': '57', 'groupId': 'BG000'}, {'value': '52', 'groupId': 'BG001'}, {'value': '109', 'groupId': 'BG002'}]}, {'title': 'At least one other known mutation', 'measurements': [{'value': '15', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}, {'title': 'Both unknown', 'measurements': [{'value': '35', 'groupId': 'BG000'}, {'value': '19', 'groupId': 'BG001'}, {'value': '54', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Cystic Fibrosis (CF) mutation', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Randomised and Treated (mITT)'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 318}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2008-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-10', 'dispFirstSubmitDate': '2012-08-06', 'completionDateStruct': {'date': '2010-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-10-06', 'studyFirstSubmitDate': '2008-02-27', 'dispFirstSubmitQcDate': '2012-08-06', 'resultsFirstSubmitDate': '2020-08-11', 'studyFirstSubmitQcDate': '2008-02-27', 'dispFirstPostDateStruct': {'date': '2012-08-15', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2020-10-09', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-10-06', 'studyFirstPostDateStruct': {'date': '2008-03-07', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-10-09', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2010-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Absolute FEV1 From Baseline Over 26 Weeks', 'timeFrame': '26 weeks', 'description': 'Change from baseline in forced expiratory volume at one second (FEV1) averaged over 26 weeks (measured at 6,14 and 26 weeks) The mean absolute change from baseline FEV1 (mL) over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach.Least square means presented are for the average change over the 6, 14, and 26 week visits.'}], 'secondaryOutcomes': [{'measure': 'Change in FEV1 From Baseline Over 26 Weeks - Dornase Users', 'timeFrame': '26 weeks', 'description': 'In the subset of dornase users, the mean absolute change from baseline FEV1 (mL) averaged over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits.\n\nChange from baseline over 26 weeks (measured at 6,14, 26 weeks) in subset of dornase users'}, {'measure': 'Rate of Protocol Defined Pulmonary Exacerbations (PDPE)', 'timeFrame': '26 weeks', 'description': 'Exacerbations treated with IV antibiotics and with at least 4 signs and symptoms according to Fuchs criteria (1994). Summary table presents the number with 0, 1,2 and 3 PDPEs during the 26 week treatment period.'}, {'measure': 'Hospitalisations Associated With Protocol Defined Pulmonary Exacerbations (PDPEs)', 'timeFrame': '26 weeks', 'description': 'The number of hospitalisations is summarised and then the rate per person is analysed.'}, {'measure': 'Antibiotic Use Associated With PDPEs', 'timeFrame': '26 weeks', 'description': 'Number of courses per person in the 26 week period is summarised and then the rate per person analysed.'}, {'measure': 'Absolute Change in FEV1 Percent Predicted at 26 Weeks', 'timeFrame': '26 weeks', 'description': 'Change from baseline at 26 weeks in FEV1 percent predicted with BOCF for those with missing values at week 26'}, {'measure': 'Change in FVC (mL) Across 26 Weeks', 'timeFrame': '26 weeks', 'description': 'Change from baseline in forced vital capacity (FVC) across 26 weeks (measured at 6,14 and 26 weeks)'}, {'measure': 'Change From Baseline FEF25-75 (mL/s) Over 26 Weeks', 'timeFrame': '26 weeks', 'description': 'Change from baseline in forced expiratory flow at 25-75% of forced vital capacity (FEF25-75) (mL/s) averaged over 26 weeks (measured at 6,14 and 26 weeks) The mean absolute change from baseline over 26 weeks (measured at week 6, 14 and 26) was compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits.'}, {'measure': 'Sputum Weight at Baseline in Response to First Dose of Treatment', 'timeFrame': 'up to 30 mins after first dose of trial treatment', 'description': 'Sputum was collected during and for 30 minutes following the administration of the first dose of study treatment.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['cystic fibrosis', 'mannitol', 'mucoactive'], 'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '16551221', 'type': 'BACKGROUND', 'citation': 'Daviskas E, Anderson SD. Hyperosmolar agents and clearance of mucus in the diseased airway. J Aerosol Med. 2006 Spring;19(1):100-9. doi: 10.1089/jam.2006.19.100.'}, {'pmid': '15691238', 'type': 'BACKGROUND', 'citation': 'Daviskas E, Anderson SD, Gomes K, Briffa P, Cochrane B, Chan HK, Young IH, Rubin BK. Inhaled mannitol for the treatment of mucociliary dysfunction in patients with bronchiectasis: effect on lung function, health status and sputum. Respirology. 2005 Jan;10(1):46-56. doi: 10.1111/j.1440-1843.2005.00659.x.'}, {'pmid': '12396422', 'type': 'BACKGROUND', 'citation': 'Daviskas E, Robinson M, Anderson SD, Bye PT. Osmotic stimuli increase clearance of mucus in patients with mucociliary dysfunction. J Aerosol Med. 2002 Fall;15(3):331-41. doi: 10.1089/089426802760292681.'}, {'pmid': '11921459', 'type': 'BACKGROUND', 'citation': 'Robinson M, Bye PT. Mucociliary clearance in cystic fibrosis. Pediatr Pulmonol. 2002 Apr;33(4):293-306. doi: 10.1002/ppul.10079.'}, {'pmid': '11171717', 'type': 'BACKGROUND', 'citation': 'Daviskas E, Anderson SD, Eberl S, Chan HK, Young IH. The 24-h effect of mannitol on the clearance of mucus in patients with bronchiectasis. Chest. 2001 Feb;119(2):414-21. doi: 10.1378/chest.119.2.414.'}, {'pmid': '10543292', 'type': 'BACKGROUND', 'citation': 'Robinson M, Daviskas E, Eberl S, Baker J, Chan HK, Anderson SD, Bye PT. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J. 1999 Sep;14(3):678-85. doi: 10.1034/j.1399-3003.1999.14c30.x.'}, {'pmid': '10351929', 'type': 'BACKGROUND', 'citation': 'Daviskas E, Anderson SD, Eberl S, Chan HK, Bautovich G. Inhalation of dry powder mannitol improves clearance of mucus in patients with bronchiectasis. Am J Respir Crit Care Med. 1999 Jun;159(6):1843-8. doi: 10.1164/ajrccm.159.6.9809074.'}, {'pmid': '9426077', 'type': 'BACKGROUND', 'citation': 'Daviskas E, Anderson SD, Brannan JD, Chan HK, Eberl S, Bautovich G. Inhalation of dry-powder mannitol increases mucociliary clearance. Eur Respir J. 1997 Nov;10(11):2449-54. doi: 10.1183/09031936.97.10112449.'}, {'pmid': '18339790', 'type': 'BACKGROUND', 'citation': 'Jaques A, Daviskas E, Turton JA, McKay K, Cooper P, Stirling RG, Robertson CF, Bye PTP, LeSouef PN, Shadbolt B, Anderson SD, Charlton B. Inhaled mannitol improves lung function in cystic fibrosis. Chest. 2008 Jun;133(6):1388-1396. doi: 10.1378/chest.07-2294. Epub 2008 Mar 13.'}, {'pmid': '32358807', 'type': 'DERIVED', 'citation': 'Nevitt SJ, Thornton J, Murray CS, Dwyer T. Inhaled mannitol for cystic fibrosis. Cochrane Database Syst Rev. 2020 May 1;5(5):CD008649. doi: 10.1002/14651858.CD008649.pub4.'}, {'pmid': '22198974', 'type': 'DERIVED', 'citation': 'Aitken ML, Bellon G, De Boeck K, Flume PA, Fox HG, Geller DE, Haarman EG, Hebestreit HU, Lapey A, Schou IM, Zuckerman JB, Charlton B; CF302 Investigators. Long-term inhaled dry powder mannitol in cystic fibrosis: an international randomized study. Am J Respir Crit Care Med. 2012 Mar 15;185(6):645-52. doi: 10.1164/rccm.201109-1666OC. Epub 2011 Dec 28.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis. Previous studies have demonstrated improvements in lung function, mucociliary clearance, changes in physical properties of mucus, 24 hour sputum weight and quality of life. The results of this study are to further investigate and confirm these findings in addition to examine the effect on antibiotic use and chest infections. It is hypothesised that inhaled mannitol will have beneficial effects compared to a control treatment. An open label phase of 26 weeks duration will follow the blinded 26 week phase. During the open label phase all subjects will receive active treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Have given written informed consent to participate in this study in accordance with local regulations\n2. Have a confirmed diagnosis of cystic fibrosis (positive sweat chloride value ≥ 60 mEq/L) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype)\n3. Be aged \\> 6 years old\n4. Have FEV1 \\>40 % and \\< 90% predicted\n5. Be able to perform all the techniques necessary to measure lung function\n\nExclusion Criteria:\n\n1. Investigators, site personnel directly affiliated with this study, or their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted.\n2. Be considered "terminally ill" or eligible for lung transplantation\n3. Have had a lung transplant\n4. Be using nebulized hypertonic saline in the 4 weeks prior to visit 1\n5. Have had a significant episode of hemoptysis (\\>60 mL) in the three months prior to enrolment\n6. Have had a myocardial infarction in the three months prior to enrolment\n7. Have had a cerebral vascular accident in the three months prior to enrolment\n8. Have had major ocular surgery in the three months prior to enrolment\n9. Have had major abdominal, chest or brain surgery in the three months prior to enrolment\n10. Have a known cerebral, aortic or abdominal aneurysm\n11. Be breast feeding or pregnant, or plan to become pregnant while in the study\n12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only)\n13. Be participating in another investigative drug study, parallel to, or within 4 weeks of visit 0\n14. Have a known allergy to mannitol\n15. Be using beta blockers\n16. Have uncontrolled hypertension - systolic BP \\> 190 and / or diastolic BP \\> 100\n17. Have a condition or be in a situation which in the Investigator\'s opinion may put the subject at significant risk, may confound results or may interfere significantly with the patient\'s participation in the study\n18. Be \'Mannitol Tolerance Test positive\'\n\n \\-'}, 'identificationModule': {'nctId': 'NCT00630812', 'briefTitle': 'Long Term Administration of Inhaled Mannitol in Cystic Fibrosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Syntara'}, 'officialTitle': 'Long Term Administration of Inhaled Mannitol in Cystic Fibrosis- A Safety and Efficacy Study', 'orgStudyIdInfo': {'id': 'DPM-CF-302'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'A', 'description': 'active treatment', 'interventionNames': ['Drug: inhaled mannitol']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'B', 'interventionNames': ['Drug: Placebo comparator']}], 'interventions': [{'name': 'inhaled mannitol', 'type': 'DRUG', 'description': '400 mg BD for 26 + 26 weeks', 'armGroupLabels': ['A']}, {'name': 'Placebo comparator', 'type': 'DRUG', 'description': 'BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase', 'armGroupLabels': ['B']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85724', 'city': 'Tucson', 'state': 'Arizona', 'country': 'United States', 'facility': 'University of Arizona', 'geoPoint': {'lat': 32.22174, 'lon': -110.92648}}, {'zip': '66106', 'city': 'Hartford', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Central Connecticut Cystic Fibrosis Center', 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '32207', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': 'Nemours Childrens Clinic', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': "Batchelor Children's Research Institute - 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