Ignite Creation Date:
2025-12-24 @ 7:35 PM
Ignite Modification Date:
2026-04-13 @ 12:48 PM
Study NCT ID:
NCT00534703
Status:
TERMINATED
Last Update Posted:
2023-02-08
First Post:
2007-09-24
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'a.lyon@imperial.ac.uk', 'phone': '+442075943409', 'title': 'Dr Alexander Lyon', 'organization': 'Imperial College London'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '6 Months', 'eventGroups': [{'id': 'EG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 1, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.', 'otherNumAtRisk': 1, 'deathsNumAtRisk': 1, 'otherNumAffected': 1, 'seriousNumAtRisk': 1, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Vessel puncture, site haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Myocarditis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Atrial Flutter', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Iron deficiency - Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Site Bleeding', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Skin Lesion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Device Alarm Issue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Product Issues', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Hyperkalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}], 'seriousEvents': [{'term': 'LVAD Alarm', 'notes': 'LVAD Alarm and power increase with hospital admission', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Product Issues', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Worsening heart failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Heart transplantation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}, {'term': 'Death', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19.1)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Safety and Feasibility of Administering AAV1/SERCA2a to LVAD Patients', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}], 'classes': [{'categories': [{'measurements': [{'value': 'NA', 'comment': 'The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.', 'groupId': 'OG000'}, {'value': 'NA', 'comment': 'The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 months', 'description': 'Safety is defined as the incidence of patients experiencing death and major adverse cardiovascular events, and out of range laboratory values. Both AAV1/SERCA2a treated cohorts (NAb+ and NAb-) will be compared to the placebo group.', 'calculatePct': False, 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and results have been presented taking a descriptive approach instead'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Exogenous Viral Vector Genome in the Myocardium Measured by qPCR for the Viral DNA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 months', 'description': 'The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Left Ventricular Function (LVEF)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.'}], 'classes': [{'categories': [{'measurements': [{'value': 'NA', 'comment': 'Note: The trial was terminated early with only 5 subjects enrolled. As a result, full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.', 'groupId': 'OG000'}, {'value': 'NA', 'comment': 'Note: The trial was terminated early with only 5 subjects enrolled. As a result, full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 months', 'description': 'Left ventricular function assessed by echocardiography and exercise capacity (6MWT, MVO2) during minimal LVAD support (low/no flow settings depending upon device) LVEF expressed as %', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Note: The trial was terminated early with only 5 subjects enrolled. As a result, full statistical analysis for both primary and secondary outcomes was impossible and results have been presented using a descriptive approach'}, {'type': 'SECONDARY', 'title': 'Levels of SERCA2a Protein', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}], 'timeFrame': '6 months', 'reportingStatus': 'POSTED', 'populationDescription': 'Data not collected due to early termination of the trial'}, {'type': 'SECONDARY', 'title': 'Other Relevant Proteins e.g. Phospholamban, the Sarcoplasmic Reticulum Calcium Release Channel, the Na+/Ca2+-Exchanger.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}], 'timeFrame': '6 months', 'reportingStatus': 'POSTED', 'populationDescription': 'Data not collected due to early termination of the trial'}, {'type': 'SECONDARY', 'title': 'Function of Isolated Myocytes', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.\n\nNo statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.\n\nJustification: The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}], 'timeFrame': '6 months', 'reportingStatus': 'POSTED', 'populationDescription': 'Data not collected due to early termination of the trial'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy\n\nAAV1/SERCA2a: AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Placebo (saline solution)\n\nPlacebo: Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '41', 'groupId': 'BG000', 'lowerLimit': '29', 'upperLimit': '69'}, {'value': '49', 'groupId': 'BG001', 'lowerLimit': '49', 'upperLimit': '49'}, {'value': '42.6', 'groupId': 'BG002', 'lowerLimit': '29', 'upperLimit': '69'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'White', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}, {'title': 'Black', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'whyStopped': 'Early termination following Trial Steering Committee recommendation', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2014-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-01', 'completionDateStruct': {'date': '2015-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-01-06', 'studyFirstSubmitDate': '2007-09-24', 'resultsFirstSubmitDate': '2018-09-17', 'studyFirstSubmitQcDate': '2007-09-24', 'lastUpdatePostDateStruct': {'date': '2023-02-08', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-02-11', 'studyFirstPostDateStruct': {'date': '2007-09-26', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-02-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2015-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Overall Safety and Feasibility of Administering AAV1/SERCA2a to LVAD Patients', 'timeFrame': '6 months', 'description': 'Safety is defined as the incidence of patients experiencing death and major adverse cardiovascular events, and out of range laboratory values. Both AAV1/SERCA2a treated cohorts (NAb+ and NAb-) will be compared to the placebo group.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Exogenous Viral Vector Genome in the Myocardium Measured by qPCR for the Viral DNA', 'timeFrame': '6 months', 'description': 'The trial was terminated early with only 5 subjects enrolled. As a result full statistical analysis for both primary and secondary outcomes was impossible and a more pragmatic approach was undertaken to assess product safety.'}, {'measure': 'Left Ventricular Function (LVEF)', 'timeFrame': '6 months', 'description': 'Left ventricular function assessed by echocardiography and exercise capacity (6MWT, MVO2) during minimal LVAD support (low/no flow settings depending upon device) LVEF expressed as %'}, {'measure': 'Levels of SERCA2a Protein', 'timeFrame': '6 months'}, {'measure': 'Other Relevant Proteins e.g. Phospholamban, the Sarcoplasmic Reticulum Calcium Release Channel, the Na+/Ca2+-Exchanger.', 'timeFrame': '6 months'}, {'measure': 'Function of Isolated Myocytes', 'timeFrame': '6 months'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Chronic Heart Failure'], 'conditions': ['Chronic Heart Failure', 'Patients That Have Received a Left Ventricular Assist Device']}, 'descriptionModule': {'briefSummary': 'The aim of the study is to determine the safety and feasibility of giving an adeno-associated viral vector expressing the sarcoplasmic reticulum calcium ATPase (SERCA2a), driven by the CMV promoter (AAV1-CMV-SERCA2a), to heart failure patients that have received a left ventricular assist device (LVAD) for an accepted clinical indication.', 'detailedDescription': 'It is a randomised, double-blind study of 24 patients that will be randomised to receive either the study drug (AAV1.SERCA2a) or placebo.\n\nThe purpose of gene transfer of SERCA2a is to improve systolic and diastolic function of the failing ventricle. Studies show that reduction of SERCA2a in failing ventricle is a key factor in depression of contraction, and that restoration of SERCA2a levels can improve function to near normal levels. The vector will be delivered during a cardiac catheterisation procedure by a 10-minute infusion into the coronary arteries.\n\nMyocardial tissue is obtained at the time of LVAD placement, as a routine part of device implantation. Further samples will be obtained when the heart is transplanted or the LVAD removed. Measures of tissue inflammation as well as efficacy of gene transfer will be made by comparing these two samples. Recovery of contractile function of the heart will be assessed during attempts to wean patients from the LVAD using standard protocols.\n\nThe results will be assessed in conjunction with two companion studies which will start earlier in the US, one performing SERCA2a gene transfer with the same vector, but delivered by direct injection into the myocardium during LVAD insertion, and one using AAV1-CMV-SERCA2a delivered percutaneously in heart failure patients. The latter has both a dose-ranging and placebo-controlled arm.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion criteria\n\n* Patients that have had a left ventricular assist device (LVAD) implanted for chronic heart failure, where chronic heart failure is defined as at least 6 months\n* Patients are clinically stable in the opinion of the clinical team looking after the patient\n* Written informed consent\n\nExclusion criteria\n\n* \\<18 or \\>70 years of age at the time of consent\n* Pregnancy or within 6 months of giving birth\n* Women of child-bearing potential not using an effective method of contraception\n* Men not using an effective method of contraception\n* Suspected or active viral, fungal or parasitic infection within 48 hours prior to administration of IMP, in the opinion of the investigator\\*.\n* Patients at a high risk of thrombosis in the opinion of the investigator\n* Patients with a previous episode of LVAD thrombosis\n* Patients with persistently raised lactate dehydrogenase (LDH \\>2.5 ULN)\n* Patients requiring triple anticoagulation i.e. warfarin and dual anti-platelet\n* Patients participating in another clinical trial\n* Patients unable to comply with the protocol mandated procedures for social or other reasons, in the opinion of the investigator and primary care physician\n\n * Eligible, enrolled and randomised patients who develop an infection will have study treatment delayed until 7 or more days after the time point when infection is no longer clinically evident.'}, 'identificationModule': {'nctId': 'NCT00534703', 'acronym': 'SERCA-LVAD', 'briefTitle': 'Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure', 'organization': {'class': 'OTHER', 'fullName': 'Imperial College London'}, 'officialTitle': 'Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure and a Left Ventricular Assist Device', 'orgStudyIdInfo': {'id': 'CRO782'}, 'secondaryIdInfos': [{'id': '2007-002809-48', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'AAV1/SERCA2A', 'description': 'SERCA gene therapy', 'interventionNames': ['Genetic: AAV1/SERCA2a']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo (saline solution)', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'AAV1/SERCA2a', 'type': 'GENETIC', 'otherNames': ['MYDICAR (R)'], 'description': 'AAV1/SERCA2a will be delivered by a percutaneous method in the catheter laboratory. Dose: 1x 10\\^13 DRP (DNase resistant particles)', 'armGroupLabels': ['AAV1/SERCA2A']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo aliquots will be of the same composition as the investigational medicinal product with the absence of the active ingredient and will be visually indistinguishable from the medicinal product. Placebo is prepared and handled exactly as above in a blinded fashion.', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'CB23 3RE', 'city': 'Cambridge', 'country': 'United Kingdom', 'facility': 'Papworth Hospital', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'zip': 'UB9 6JH', 'city': 'Middlesex', 'country': 'United Kingdom', 'facility': 'Harefield Hospital, Royal Brompton and Harefiled NHS Trust', 'geoPoint': {'lat': 51.53174, 'lon': -0.26856}}], 'overallOfficials': [{'name': 'Sian Harding', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Imperial College London'}, {'name': 'Alexander Lyon', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Imperial College London'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Imperial College London', 'class': 'OTHER'}, 'collaborators': [{'name': 'British Heart Foundation', 'class': 'OTHER'}, {'name': 'Leducq Foundation', 'class': 'OTHER'}, {'name': 'Celladon Corporation', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}