Ignite Creation Date:
2025-12-24 @ 5:41 PM
Ignite Modification Date:
2026-02-28 @ 11:20 AM
Study NCT ID:
NCT03129568
Status:
COMPLETED
Last Update Posted:
2021-11-26
First Post:
2017-04-14
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002311', 'term': 'Cardiomyopathy, Dilated'}], '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': 'hidemasa@okayama-u.ac.jp', 'phone': '086-235-6506', 'title': 'Dr. Hidemasa Oh', 'phoneExt': '6506', 'organization': 'Okayama University Hospital'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'Single arm study without placebo control comparator'}}, 'adverseEventsModule': {'timeFrame': '1 year', 'eventGroups': [{'id': 'EG000', 'title': 'CDC Infusion', 'description': 'CDCs infusion by coronary intervention.\n\nCDC infusion: Injection of CDCs (0.3 million per kg of body weight).', 'otherNumAtRisk': 5, 'deathsNumAtRisk': 5, 'otherNumAffected': 0, 'seriousNumAtRisk': 5, 'deathsNumAffected': 1, 'seriousNumAffected': 1}], 'seriousEvents': [{'term': 'heart failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'ischemic event', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CDC Infusion', 'description': 'CDCs infusion by coronary intervention.\n\nCDC infusion: Injection of CDCs (0.3 million per kg of body weight).'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 months after CDC treatment', 'description': 'Assessment of major adverse cardiac events include death, sustained/symptomatic ventricular tachycardia, aggravation of heart failure, acute coronary syndrome, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Adverse events'}, {'type': 'SECONDARY', 'title': 'Change in Ejection Fraction', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'CDC Infusion', 'description': 'CDCs infusion by coronary intervention.\n\nCDC infusion: Injection of CDCs (0.3 million per kg of body weight).'}], 'classes': [{'categories': [{'measurements': [{'value': '4.5', 'spread': '10.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '6 months after protocol treatment', 'description': 'To determine the changes in cardiac function by ejection fraction with cardiac MRI evaluation in CDC-treated participants from baseline to 6 months of follow up.', 'unitOfMeasure': 'percentage of ejection fraction', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Changes in cardiac function'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'CDC Infusion', 'description': 'CDCs infusion by coronary intervention.\n\nCDC infusion: Injection of CDCs (0.3 million per kg of body weight).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'CDC Infusion', 'description': 'CDCs infusion by coronary intervention.\n\nCDC infusion: Injection of CDCs (0.3 million per kg of body weight).'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '11.4', 'spread': '6.7', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Cardiac Function', 'classes': [{'categories': [{'measurements': [{'value': '28.5', 'spread': '10.7', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'percentage of baseline ejection fraction', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Consecutive enrollment of 5 patients to receive CDC infusion'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-12-09', 'size': 106566, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2021-09-30T23:33', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Transcoronary infusion'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-04-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-11', 'completionDateStruct': {'date': '2020-12-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-11-23', 'studyFirstSubmitDate': '2017-04-14', 'resultsFirstSubmitDate': '2021-09-30', 'studyFirstSubmitQcDate': '2017-04-23', 'lastUpdatePostDateStruct': {'date': '2021-11-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-09-30', 'studyFirstPostDateStruct': {'date': '2017-04-26', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2021-11-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-09-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.', 'timeFrame': '6 months after CDC treatment', 'description': 'Assessment of major adverse cardiac events include death, sustained/symptomatic ventricular tachycardia, aggravation of heart failure, acute coronary syndrome, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.'}], 'secondaryOutcomes': [{'measure': 'Change in Ejection Fraction', 'timeFrame': '6 months after protocol treatment', 'description': 'To determine the changes in cardiac function by ejection fraction with cardiac MRI evaluation in CDC-treated participants from baseline to 6 months of follow up.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Dilated Cardiomyopathy']}, 'referencesModule': {'references': [{'pmid': '28052915', 'type': 'BACKGROUND', 'citation': 'Ishigami S, Ohtsuki S, Eitoku T, Ousaka D, Kondo M, Kurita Y, Hirai K, Fukushima Y, Baba K, Goto T, Horio N, Kobayashi J, Kuroko Y, Kotani Y, Arai S, Iwasaki T, Sato S, Kasahara S, Sano S, Oh H. Intracoronary Cardiac Progenitor Cells in Single Ventricle Physiology: The PERSEUS (Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease) Randomized Phase 2 Trial. Circ Res. 2017 Mar 31;120(7):1162-1173. doi: 10.1161/CIRCRESAHA.116.310253. Epub 2017 Jan 4.'}, {'pmid': '26232942', 'type': 'BACKGROUND', 'citation': 'Tarui S, Ishigami S, Ousaka D, Kasahara S, Ohtsuki S, Sano S, Oh H. Transcoronary infusion of cardiac progenitor cells in hypoplastic left heart syndrome: Three-year follow-up of the Transcoronary Infusion of Cardiac Progenitor Cells in Patients With Single-Ventricle Physiology (TICAP) trial. J Thorac Cardiovasc Surg. 2015 Nov;150(5):1198-1207, 1208.e1-2. doi: 10.1016/j.jtcvs.2015.06.076. Epub 2015 Jul 8.'}, {'pmid': '25403163', 'type': 'BACKGROUND', 'citation': 'Ishigami S, Ohtsuki S, Tarui S, Ousaka D, Eitoku T, Kondo M, Okuyama M, Kobayashi J, Baba K, Arai S, Kawabata T, Yoshizumi K, Tateishi A, Kuroko Y, Iwasaki T, Sato S, Kasahara S, Sano S, Oh H. Intracoronary autologous cardiac progenitor cell transfer in patients with hypoplastic left heart syndrome: the TICAP prospective phase 1 controlled trial. Circ Res. 2015 Feb 13;116(4):653-64. doi: 10.1161/CIRCRESAHA.116.304671. Epub 2014 Nov 17.'}, {'pmid': '33298561', 'type': 'RESULT', 'citation': 'Hirai K, Ousaka D, Fukushima Y, Kondo M, Eitoku T, Shigemitsu Y, Hara M, Baba K, Iwasaki T, Kasahara S, Ohtsuki S, Oh H. Cardiosphere-derived exosomal microRNAs for myocardial repair in pediatric dilated cardiomyopathy. Sci Transl Med. 2020 Dec 9;12(573):eabb3336. doi: 10.1126/scitranslmed.abb3336.'}]}, 'descriptionModule': {'briefSummary': 'A phase 1 prospective study to determine the procedural feasibility and safety and preliminary efficacy of intracoronary infusion of cardiosphere-derived cells (CDCs) in patients with dilated cardiomyopathy.', 'detailedDescription': 'Five consecutive patients will be enrolled to a phase 1 study to verify the procedural feasibility and safety with the CDC infusion group.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients diagnosed as dilated cardiomyopathy.\n* Patients aged under 18 years old.\n* Cardiac ejection fraction \\< 40%.\n\nExclusion Criteria:\n\n* Contradiction to cardiac magnetic resonance imaging.\n* Cardiogenic shock.\n* A patient with unstoppable extracorporeal circulation.\n* A patient with lethal, uncontrollable arrhythmia.\n* A patient with a complication of coronary artery disease.\n* A patient with a complication of brain dysfunction due to circulatory failure.\n* A patient with malignant neoplasm.\n* A patient with a complication of a serious neurologic disorder.\n* A patient with high-grade pulmonary embolism or pulmonary hypertension.\n* A patient with high-grade renal failure.\n* A patient with multiple organ failure.\n* Active infection (including endocarditis).\n* Sepsis.\n* Active hemorrhagic disease (e.g. gastrointestinal bleeding, injury).'}, 'identificationModule': {'nctId': 'NCT03129568', 'acronym': 'TICAP-DCM', 'briefTitle': 'Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy', 'organization': {'class': 'OTHER', 'fullName': 'Okayama University'}, 'officialTitle': 'A Prospective Phase 1 Trial of Cardiac Progenitor Cell Therapy in Children With Dilated Cardiomyopathy', 'orgStudyIdInfo': {'id': '01F1701003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'CDC infusion', 'description': 'CDCs infusion by coronary intervention.', 'interventionNames': ['Biological: CDC infusion']}], 'interventions': [{'name': 'CDC infusion', 'type': 'BIOLOGICAL', 'description': 'Injection of CDCs (0.3 million per kg of body weight).', 'armGroupLabels': ['CDC infusion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '700-8558', 'city': 'Okayama', 'country': 'Japan', 'facility': 'Okayama University Hospital', 'geoPoint': {'lat': 34.65, 'lon': 133.93333}}], 'overallOfficials': [{'name': 'Shigeru Uemura, M.D., Ph.D.', 'role': 'STUDY_CHAIR', 'affiliation': 'Shonan Tobu Clinic'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Okayama University', 'class': 'OTHER'}, 'collaborators': [{'name': 'Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Hidemasa Oh, MD', 'investigatorAffiliation': 'Okayama University'}}}}