Ignite Creation Date:
2025-12-25 @ 12:44 AM
Ignite Modification Date:
2025-12-31 @ 5:01 PM
Study NCT ID:
NCT00789867
Status:
COMPLETED
Last Update Posted:
2020-01-18
First Post:
2008-11-11
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Single Dose of pGM169/GL67A in CF Patients
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': 's.soussi@imperial.ac.uk', 'phone': '0207 594 7980', 'title': 'Samia Soussi', 'organization': 'Imperial College'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '28 days', 'eventGroups': [{'id': 'EG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer', 'otherNumAtRisk': 17, 'deathsNumAtRisk': 17, 'otherNumAffected': 14, 'seriousNumAtRisk': 17, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer', 'otherNumAtRisk': 10, 'deathsNumAtRisk': 10, 'otherNumAffected': 7, 'seriousNumAtRisk': 10, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 1, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Mild self limiting influenza like systemic response', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 14, 'numAffected': 14}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG002', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}], 'seriousEvents': [{'term': 'Acute pancreatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}, {'term': 'Injury in luvula sustained from pressure of tube during bronchoscopy 03/04/2009', 'stats': [{'groupId': 'EG000', 'numAtRisk': 17, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 10, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Body Maximum Temperature', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer'}, {'id': 'OG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'OG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}], 'classes': [{'categories': [{'measurements': [{'value': '38.6', 'spread': '0.5', 'groupId': 'OG000'}, {'value': '38.0', 'spread': '0.7', 'groupId': 'OG001'}, {'value': '37.4', 'spread': '0.3', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.0002', 'groupIds': ['OG000', 'OG001', 'OG002'], 'statisticalMethod': 'Kruskal-Wallis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '6-8h', 'unitOfMeasure': 'celsius', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'A lower number of participants due to missing data'}, {'type': 'PRIMARY', 'title': 'Blood Leukocytes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer'}, {'id': 'OG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'OG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}], 'classes': [{'categories': [{'measurements': [{'value': '15.8', 'spread': '3.2', 'groupId': 'OG000'}, {'value': '14.1', 'spread': '4.5', 'groupId': 'OG001'}, {'value': '12.8', 'spread': '3.8', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.22', 'groupIds': ['OG000', 'OG001', 'OG002'], 'statisticalMethod': 'Kruskal-Wallis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '8h', 'description': 'Blood leukocytes measure', 'unitOfMeasure': 'x1000000000 cells/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'A lower number of participants due to missing data'}, {'type': 'PRIMARY', 'title': 'Blood Neutrophils', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer'}, {'id': 'OG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'OG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}], 'classes': [{'categories': [{'measurements': [{'value': '13.9', 'spread': '3.4', 'groupId': 'OG000'}, {'value': '11.3', 'spread': '4.1', 'groupId': 'OG001'}, {'value': '9.8', 'spread': '3.5', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.06', 'groupIds': ['OG000', 'OG001', 'OG002'], 'statisticalMethod': 'Kruskal-Wallis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '8h', 'description': 'Blood neutrophils measures', 'unitOfMeasure': '1000000000/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'A lower number of participants due to missing data'}, {'type': 'PRIMARY', 'title': 'FEV1 Relative % Drop', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer'}, {'id': 'OG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'OG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}], 'classes': [{'categories': [{'measurements': [{'value': '24.6', 'spread': '9.3', 'groupId': 'OG000'}, {'value': '17.5', 'spread': '7.8', 'groupId': 'OG001'}, {'value': '16.8', 'spread': '4.0', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.08', 'groupIds': ['OG000', 'OG001', 'OG002'], 'statisticalMethod': 'Kruskal-Wallis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '8h', 'description': 'FEV1 relative % drop measure', 'unitOfMeasure': '% of drop', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'A lower number of participants due to missing data'}, {'type': 'PRIMARY', 'title': 'FVC Relative % Drop', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer'}, {'id': 'OG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'OG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}], 'classes': [{'categories': [{'measurements': [{'value': '20.7', 'spread': '2.9', 'groupId': 'OG000'}, {'value': '13.7', 'spread': '2.2', 'groupId': 'OG001'}, {'value': '14.7', 'spread': '2.2', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.22', 'groupIds': ['OG000', 'OG001', 'OG002'], 'statisticalMethod': 'Kruskal-Wallis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '6h', 'description': 'FVC relative % drop measure', 'unitOfMeasure': '% drop', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'A lower number of participants due to missing data'}, {'type': 'PRIMARY', 'title': 'Lung Clearance Index - LCI', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer nebulizer'}, {'id': 'OG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'OG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}], 'classes': [{'categories': [{'measurements': [{'value': '0.75', 'spread': '0.3', 'groupId': 'OG000'}, {'value': '0.32', 'spread': '0.1', 'groupId': 'OG001'}, {'value': '0.32', 'spread': '.01', 'groupId': 'OG002'}]}]}], 'analyses': [{'pValue': '0.003', 'groupIds': ['OG000', 'OG001', 'OG002'], 'statisticalMethod': 'Kruskal-Wallis', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '8h', 'description': 'Lung clearance index measure is a measure of abnormal ventilation distribution derived from the multiple breath inert gas washout technique.', 'unitOfMeasure': 'index', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'A lower number of participants due to missing data'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': '20 ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer'}, {'id': 'FG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'FG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}, {'groupId': 'FG001', 'numSubjects': '10'}, {'groupId': 'FG002', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '17'}, {'groupId': 'FG001', 'numSubjects': '10'}, {'groupId': 'FG002', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '17', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}, {'value': '35', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer'}, {'id': 'BG001', 'title': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer'}, {'id': 'BG002', 'title': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}, {'value': '33', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '26.7', 'groupId': 'BG000', 'lowerLimit': '17.3', 'upperLimit': '50.1'}, {'value': '33.2', 'groupId': 'BG001', 'lowerLimit': '16.4', 'upperLimit': '61.6'}, {'value': '32.6', 'groupId': 'BG002', 'lowerLimit': '24.3', 'upperLimit': '46.4'}, {'value': '27.4', 'groupId': 'BG003', 'lowerLimit': '16.4', 'upperLimit': '61.6'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '12', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}, {'value': '23', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}, {'value': '35', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 35}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2008-11', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-01', 'completionDateStruct': {'date': '2010-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-01-09', 'studyFirstSubmitDate': '2008-11-11', 'resultsFirstSubmitDate': '2019-05-23', 'studyFirstSubmitQcDate': '2008-11-11', 'lastUpdatePostDateStruct': {'date': '2020-01-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-01-09', 'studyFirstPostDateStruct': {'date': '2008-11-13', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-01-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2009-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Body Maximum Temperature', 'timeFrame': '6-8h'}, {'measure': 'Blood Leukocytes', 'timeFrame': '8h', 'description': 'Blood leukocytes measure'}, {'measure': 'Blood Neutrophils', 'timeFrame': '8h', 'description': 'Blood neutrophils measures'}, {'measure': 'FEV1 Relative % Drop', 'timeFrame': '8h', 'description': 'FEV1 relative % drop measure'}, {'measure': 'FVC Relative % Drop', 'timeFrame': '6h', 'description': 'FVC relative % drop measure'}, {'measure': 'Lung Clearance Index - LCI', 'timeFrame': '8h', 'description': 'Lung clearance index measure is a measure of abnormal ventilation distribution derived from the multiple breath inert gas washout technique.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Single dose', 'Pilot', 'Safety', 'Gene expression', 'Tolerability', 'CFTR gene', 'Cystic fibrosis', 'Non-viral'], 'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '10459902', 'type': 'BACKGROUND', 'citation': 'Alton EW, Stern M, Farley R, Jaffe A, Chadwick SL, Phillips J, Davies J, Smith SN, Browning J, Davies MG, Hodson ME, Durham SR, Li D, Jeffery PK, Scallan M, Balfour R, Eastman SJ, Cheng SH, Smith AE, Meeker D, Geddes DM. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet. 1999 Mar 20;353(9157):947-54. doi: 10.1016/s0140-6736(98)06532-5.'}, {'pmid': '26623687', 'type': 'RESULT', 'citation': 'Alton EW, Boyd AC, Porteous DJ, Davies G, Davies JC, Griesenbach U, Higgins TE, Gill DR, Hyde SC, Innes JA; UK Cystic Fibrosis Gene Therapy Consortium *. A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial. Am J Respir Crit Care Med. 2015 Dec 1;192(11):1389-92. doi: 10.1164/rccm.201506-1193LE. No abstract available.'}], 'seeAlsoLinks': [{'url': 'http://www.cfgenetherapy.org.uk/', 'label': 'UK CF Gene Therapy Consortium website'}]}, 'descriptionModule': {'briefSummary': 'The study objectives are to assess safety, tolerability and gene expression after a single dose of non-viral CFTR gene therapy (pGM169/GL67A) administered to the nose and lungs of patients with cystic fibrosis.', 'detailedDescription': 'The trial is designed as single administration to nose and lung. Initially, in Part A, 3 patients will be dosed individually one week apart with 10 ml (26.5mg pDNA) via nebuliser and a nasal dose equivalent to 10% of this (based on relative surface area calculations: conducting airways approximate 540 cm2; nasal epithelium from both nostrils approximately 40 cm2). Based on our previous study, we do not expect any side effects of the nasal dose, but we are taking this opportunity, as this group will not be undergoing bronchoscopic efficacy measures, to assess gene transfer to the nasal epithelium. A further group of 3 patients will then be treated in exactly the same way with 20 ml nebulised and a 2 ml nasal dose.\n\nSubsequently, patients will receive doses of 2 ml (nasal) and 20 ml (nebulised). These patients will undergo more intensive monitoring for gene expression both before and after administration.\n\nIn Part B of the protocol, we will test combinations of delivery conditions and dose in an attempt to identify the maximal tolerated dose. We may also use Ibuprofen or Prednisolone in standard clinical doses around the dosing period to reduce the inflammatory response. Delivery conditions include: standard nebulisation (each 5 ml over 25 minutes as in Part A), slow (each 5 ml over 75-150 minutes) and divided (standard rate delivery with a period of up to 6 hours between aliquots). With these conditions we will test the following doses until a tolerable dose is reached: 20 ml (no standard delivery as sufficient data already available from Part A); 10 ml; 5 ml; 2.5 ml. Each dosing strategy will initially be performed in a cohort of 3 patients although numbers may need to be increased to 6 if data are inconclusive. Once a satisfactory Single dose of pGM169/GL67A in CF patients; cro851 Version 10; 16.08.2010 10 dose and nebulisation strategy has been identified, the numbers receiving this will be increased to 6. The maximum number of patients recruited to this arm of the study will be 30. Part B will also allow either these subjects or others to receive a 2 ml nasal dose with both pre and post-measurements of nasal PD.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Cystic fibrosis confirmed by sweat testing or genetic analysis\n* Males and females aged 16 years and above\n* Forced expiratory volume in the 1st second (FEV1) \\> 60% predicted values\n* Clinical stability at entry\n* Prepared to take effective contraceptive precautions for the duration of their participation in the study and for 3 months thereafter\n* If taking regular rhDNase (pulmozyme) is willing, and considered able by independent medical carers, to withhold treatment for 24 hours before and 24 hours after the gene therapy dose\n* Written informed consent obtained\n* Permission to inform GP of participation in study\n\nExclusion Criteria:\n\n* Infection with Burkholderia cepacia complex organisms or MRSA\n* Significant nasal pathology including polyps, clinically-significant rhinosinusitis, or recurrent severe epistaxis (nose bleeds)\n* Acute upper respiratory tract infection within the last 2 weeks\n* Previous spontaneous pneumothorax without pleurodesis\n* Recurrent severe haemoptysis\n* Current smoker\n* Significant comorbidity including:\n\n 1. Moderate/severe CF liver disease\n 2. Significant renal impairment\n 3. Significant coagulopathy\n* Receiving 2nd line immunosuppressant drugs such as methotrexate, cyclosporine, intravenous immunoglobulin preparations\n* Pregnant or breastfeeding'}, 'identificationModule': {'nctId': 'NCT00789867', 'briefTitle': 'Single Dose of pGM169/GL67A in CF Patients', 'organization': {'class': 'OTHER', 'fullName': 'Imperial College London'}, 'officialTitle': 'Evaluation of Safety and Gene Expression With a Single Dose of pGM169/GL67A Administered to the Nose and Lung of Individuals With Cystic Fibrosis', 'orgStudyIdInfo': {'id': 'cro851'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '20ml pGM169/GL67A', 'description': 'Received a nebulized dose 20ml via an breath-actuated nebulizer', 'interventionNames': ['Drug: pGM169/GL67A']}, {'type': 'EXPERIMENTAL', 'label': '10ml pGM169/GL67A', 'description': 'Received a nebulized dose 10ml via an breath-actuated nebulizer', 'interventionNames': ['Drug: pGM169/GL67A']}, {'type': 'EXPERIMENTAL', 'label': '5ml pGM169/GL67A', 'description': 'Received a nebulized dose 5ml via an breath-actuated nebulizer', 'interventionNames': ['Drug: pGM169/GL67A']}], 'interventions': [{'name': 'pGM169/GL67A', 'type': 'DRUG', 'otherNames': ['5ml pGM169/GL67A, 10ml pGM169/GL67A and 20ml pGM169/GL67A'], 'description': 'Received a nebulized dose via an breath-actuated nebulizer', 'armGroupLabels': ['10ml pGM169/GL67A', '20ml pGM169/GL67A', '5ml pGM169/GL67A']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'SW3 6NP', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Brompton Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Eric Alton', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Imperial College London'}, {'name': 'Jane C Davies', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Imperial College London'}, {'name': 'Uta Griesenbach', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Imperial College London'}, {'name': 'Steve Hyde', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Oxford'}, {'name': 'Deborah Gill', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Oxford'}, {'name': 'David Porteous', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Edinburgh University'}, {'name': 'Chris Boyd', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Edinburgh University'}, {'name': 'Alastair Innes', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Edinburgh University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Imperial College London', 'class': 'OTHER'}, 'collaborators': [{'name': 'Royal Brompton & Harefield NHS Foundation Trust', 'class': 'OTHER'}, {'name': 'University of Oxford', 'class': 'OTHER'}, {'name': 'University of Edinburgh', 'class': 'OTHER'}, {'name': 'Cystic Fibrosis Trust', 'class': 'OTHER'}, {'name': 'University of Pennsylvania', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}