Ignite Creation Date:
2025-12-25 @ 4:51 AM
Ignite Modification Date:
2026-01-26 @ 5:36 PM
Study NCT ID:
NCT00629018
Status:
COMPLETED
Last Update Posted:
2015-05-12
First Post:
2008-02-25
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Efficacy Study of Stem Cell Transplantation to Treat Dilated Cardiomyopathy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002311', 'term': 'Cardiomyopathy, Dilated'}, {'id': 'D006333', 'term': 'Heart Failure'}], 'ancestors': [{'id': 'D006332', 'term': 'Cardiomegaly'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D009202', 'term': 'Cardiomyopathies'}, {'id': 'D000083083', 'term': 'Laminopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'bvrtovec@stanford.edu', 'phone': '+3861 522 2844', 'title': 'Prof. Dr. Bojan Vrtovec', 'organization': 'UMC Ljubljana'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'SC Group', 'description': '55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability', 'otherNumAtRisk': 55, 'otherNumAffected': 2, 'seriousNumAtRisk': 55, 'seriousNumAffected': 8}, {'id': 'EG001', 'title': 'Control Group', 'description': '55 patients were randomized to standard medical therapy, without stem cell injection.', 'otherNumAtRisk': 55, 'otherNumAffected': 0, 'seriousNumAtRisk': 55, 'seriousNumAffected': 19}], 'otherEvents': [{'term': 'Non-sustained ventricular tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 55, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 55, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Death', 'stats': [{'groupId': 'EG000', 'numAtRisk': 55, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 55, 'numAffected': 19}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '2'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Heart Failure Mortality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'SC Group', 'description': '55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability'}, {'id': 'OG001', 'title': 'Control Group', 'description': '55 patients were randomized to standard medical therapy, without stem cell injection.'}], 'classes': [{'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}, {'value': '19', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.01', 'groupIds': ['OG000', 'OG001'], 'ciPctValue': '95', 'groupDescription': 'The minimal sample size for the study was calculated using a pre-specified power of 90% and P value of 0.05.', 'statisticalMethod': 'Log Rank', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'NUMBER', 'timeFrame': '5 years', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The minimal sample size for the study was calculated using a pre-specified power of 90% and P value of 0.05.'}, {'type': 'PRIMARY', 'title': 'Changes in Left Ventricular Ejection Fraction', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}, {'value': '55', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'SC Group', 'description': '55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability'}, {'id': 'OG001', 'title': 'Control Group', 'description': '55 patients were randomized to standard medical therapy, without stem cell injection.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '24.3', 'spread': '6.5', 'groupId': 'OG000'}, {'value': '25.7', 'spread': '4.1', 'groupId': 'OG001'}]}]}, {'title': '5 years', 'categories': [{'measurements': [{'value': '30.0', 'spread': '5.1', 'groupId': 'OG000'}, {'value': '23.3', 'spread': '4.2', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '5 years', 'description': 'Left ventricular ejection fraction measured by echocardiography', 'unitOfMeasure': 'Percentage of ejection', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Changes in Exercise Capacity', 'timeFrame': '5 years', 'reportingStatus': 'NOT_POSTED'}, {'type': 'SECONDARY', 'title': 'Changes in Electrophysiologic Properties of Ventricular Myocardium', 'timeFrame': '6 months', 'reportingStatus': 'NOT_POSTED'}, {'type': 'SECONDARY', 'title': 'Changes in Plasma Inflammatory Markers', 'timeFrame': '6 months', 'reportingStatus': 'NOT_POSTED'}, {'type': 'SECONDARY', 'title': 'Changes in Left Ventricular Function', 'timeFrame': '5 years', 'reportingStatus': 'NOT_POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'SC Group', 'description': '55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability'}, {'id': 'FG001', 'title': 'Control Group', 'description': '55 patients were randomized to standard medical therapy, without stem cell injection.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '55'}, {'groupId': 'FG001', 'numSubjects': '55'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '55'}, {'groupId': 'FG001', 'numSubjects': '55'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'This study consisted of an open-label randomized study design conducted at the Advanced Heart Failure and Transplantation Center at University Medical Center Ljubljana in collaboration with the Methodist DeBakey Heart Center and Stanford University.', 'preAssignmentDetails': 'Patients with acute multi-organ failure or history of haematologic neoplasms were not included.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'BG000'}, {'value': '55', 'groupId': 'BG001'}, {'value': '110', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'SC Group', 'description': '55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability'}, {'id': 'BG001', 'title': 'Control Group', 'description': '55 patients were randomized to standard medical therapy, without stem cell injection.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '53', 'groupId': 'BG000'}, {'value': '52', 'groupId': 'BG001'}, {'value': '105', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '53', 'spread': '8', 'groupId': 'BG000'}, {'value': '55', 'spread': '7', 'groupId': 'BG001'}, {'value': '54', 'spread': '9', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '21', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '45', 'groupId': 'BG000'}, {'value': '44', 'groupId': 'BG001'}, {'value': '89', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Slovenia', 'categories': [{'measurements': [{'value': '55', 'groupId': 'BG000'}, {'value': '55', 'groupId': 'BG001'}, {'value': '110', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 110}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-04', 'completionDateStruct': {'date': '2013-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-04-23', 'studyFirstSubmitDate': '2008-02-25', 'resultsFirstSubmitDate': '2013-04-09', 'studyFirstSubmitQcDate': '2008-02-25', 'lastUpdatePostDateStruct': {'date': '2015-05-12', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2015-04-23', 'studyFirstPostDateStruct': {'date': '2008-03-05', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-05-12', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Heart Failure Mortality', 'timeFrame': '5 years'}, {'measure': 'Changes in Left Ventricular Ejection Fraction', 'timeFrame': '5 years', 'description': 'Left ventricular ejection fraction measured by echocardiography'}], 'secondaryOutcomes': [{'measure': 'Changes in Exercise Capacity', 'timeFrame': '5 years'}, {'measure': 'Changes in Electrophysiologic Properties of Ventricular Myocardium', 'timeFrame': '6 months'}, {'measure': 'Changes in Plasma Inflammatory Markers', 'timeFrame': '6 months'}, {'measure': 'Changes in Left Ventricular Function', 'timeFrame': '5 years'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Stem Cells', 'Heart Failure', 'Dilated Cardiomyopathy'], 'conditions': ['Dilated Cardiomyopathy']}, 'referencesModule': {'references': [{'pmid': '21440864', 'type': 'DERIVED', 'citation': 'Vrtovec B, Poglajen G, Sever M, Lezaic L, Domanovic D, Cernelc P, Haddad F, Torre-Amione G. Effects of intracoronary stem cell transplantation in patients with dilated cardiomyopathy. J Card Fail. 2011 Apr;17(4):272-81. doi: 10.1016/j.cardfail.2010.11.007. Epub 2010 Dec 24.'}]}, 'descriptionModule': {'briefSummary': 'Several studies have documented that transplantation of bone marrow-derived cells (BMC) following acute myocardial infarction is associated with a reduction in infarct scar size and improvements in left ventricular function and perfusion. The available evidence in humans suggests that BMC transplantation is associated with improvements in physiologic and anatomic parameters in both acute myocardial infarction and chronic ischemic heart disease, above and beyond the conventional therapy. In particular, intracoronary application of BMC is proved to be safe and was associated with significant improvement in the left ventricular ejection fraction (LVEF) in patients with chronic heart failure.\n\nIn contrast to ischemic heart failure, the data on effects of BMC transplantation in patients with dilated cardiomyopathy are limited to pre-clinical studies. In a rat model of dilated cardiomyopathy, intramyocardial delivery of pluripotent mesenchymal cells improved LVEF, possibly through induction of myogenesis and angiogenesis, as well as by inhibition of myocardial fibrosis, suggesting that the beneficial effects of stem cell transplantation in dilated cardiomyopathy may primarily be related to their ability to supply large amounts of angiogenic, antiapoptotic, and mitogenic factors. Similarly, transplantation of cocultured mesenchymal stem cells and skeletal myoblasts was shown to improve LVEF in a murine model of Chagas disease.\n\nStudy Aim:\n\nTo define the clinical effects of BMC transplantation in dilated cardiomyopathy in a pilot clinical study investigating the effects of intracoronary CD34+ cell transplantation on functional, structural, neurohormonal, and electrophysiologic parameters in patients with end-stage dilated cardiomyopathy.', 'detailedDescription': 'Patients were randomly allocated in a 1:1 ratio to receive intracoronary transplantation of autologous CD34+ stem cells (SC group) or no intracoronary infusion (control group). At the time of enrollment, and at yearly intervals thereafter, we performed detailed clinical evaluation, echocardiography, 6-minute walk test, and measured plasma levels of NT-proBNP. To better-define the potential role of inflammatory response, we also measured plasma inflammatory markers (tumor necrosis factor \\[TNF\\]-α and interleukin \\[IL\\]-6) at the time of CD34+ stem cell injection.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Normal coronary angiogram\n* Left ventricular ejection fraction \\< 40%\n* NYHA III or IV heart failure symptoms\n* Bone marrow reactivity (G-CSF test)\n* Presence of viable myocardium\n\nExclusion Criteria:\n\n* Hematologic malignancy\n* Multiorgan failure'}, 'identificationModule': {'nctId': 'NCT00629018', 'briefTitle': 'Safety and Efficacy Study of Stem Cell Transplantation to Treat Dilated Cardiomyopathy', 'organization': {'class': 'OTHER', 'fullName': 'University Medical Centre Ljubljana'}, 'officialTitle': 'The Effects of Autologous Intracoronary Stem Cell Transplantation In Patients With End-Stage Dilated Cardiomyopathy', 'orgStudyIdInfo': {'id': 'DCM-SCT1'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'SC Group', 'description': "SC therapy,'Bone Marrow Stimulation','CD34+ autologous stem cell transplantation':\n\nIn the SC group, CD34+ cells were mobilized by granulocyte colony-stimulating factor and collected via apheresis. Patients underwent myocardial scintigraphy and cells were injected in the artery supplying segments with the greatest perfusion defect", 'interventionNames': ['Biological: CD34+ autologous stem cell transplantation', 'Drug: Bone Marrow Stimulation', 'Biological: SC therapy']}, {'type': 'NO_INTERVENTION', 'label': 'Controls', 'description': 'Patients receiving no cell therapy.'}], 'interventions': [{'name': 'CD34+ autologous stem cell transplantation', 'type': 'BIOLOGICAL', 'description': 'Peripheral blood stem cells will be mobilized by daily subcutaneous injections of filgrastim; CD34+ cells will be collected via apheresis and labeled with technetium. Patients will undergo myocardial perfusion scintigraphy for myocardial viability assessment and the collected CD34+ cells will be injected intracoronary in the artery supplying the segments of reduced tracer accumulation', 'armGroupLabels': ['SC Group']}, {'name': 'Bone Marrow Stimulation', 'type': 'DRUG', 'otherNames': ['G-CSF stimulation'], 'description': 'Patients will undergo filgrastim stimulation and viability assessment using the same protocol as in Arm 1. However, in this group, no intracoronary stem cell delivery will be performed; the patients will receive placebo (saline).', 'armGroupLabels': ['SC Group']}, {'name': 'SC therapy', 'type': 'BIOLOGICAL', 'description': 'In the SC group, CD34+ cells were mobilized by granulocyte colony-stimulating factor and collected via apheresis. Patients underwent myocardial scintigraphy and cells were injected in the artery supplying segments with the greatest perfusion defect', 'armGroupLabels': ['SC Group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '1000', 'city': 'Ljubljana', 'country': 'Slovenia', 'facility': 'Ljubljana University Medical Center', 'geoPoint': {'lat': 46.05108, 'lon': 14.50513}}], 'overallOfficials': [{'name': 'Guillermo Torre Amione, MD, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Methodist DeBakey Heart Center, Houston TX, USA'}, {'name': 'Francois Haddad, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Stanford University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Medical Centre Ljubljana', 'class': 'OTHER'}, 'collaborators': [{'name': 'Blood Transfusion Centre of Slovenia', 'class': 'OTHER_GOV'}, {'name': 'Stanford University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'prof. dr. Bojan vrtovec', 'investigatorFullName': 'Bojan Vrtovec', 'investigatorAffiliation': 'University Medical Centre Ljubljana'}}}}