Ignite Creation Date:
2025-12-25 @ 5:19 AM
Ignite Modification Date:
2025-12-26 @ 4:27 AM
Study NCT ID:
NCT03409627
Status:
COMPLETED
Last Update Posted:
2020-12-04
First Post:
2017-11-03
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Novel INXN-4001 Triple Effector Plasmid in Heart Failure
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006333', 'term': 'Heart Failure'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}], 'ancestors': [{'id': 'D006331', 'term': 'Heart Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-04-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-12', 'completionDateStruct': {'date': '2020-08-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-12-03', 'studyFirstSubmitDate': '2017-11-03', 'studyFirstSubmitQcDate': '2018-01-17', 'lastUpdatePostDateStruct': {'date': '2020-12-04', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-01-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-03-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To evaluate safety and feasibility of INXN-4001', 'timeFrame': '6 months', 'description': 'As assessed by incidence of all study intervention-related adverse events occurring up to 6 months post- treatment intervention-related adverse events'}], 'secondaryOutcomes': [{'measure': 'Wean tolerability following treatment with INXN-4001', 'timeFrame': '12 months', 'description': 'Improvement in 6 minute walk duration and distance'}, {'measure': 'Quality of Life following treatment with INXN-4001', 'timeFrame': '12 months', 'description': 'Change in Kansas City Cardiomyopathy Questionnaire responses'}, {'measure': 'Feasibility of biosensor activity tracking', 'timeFrame': '12 months', 'description': 'Ability to collect daily activity level as measured by wearable biosensor'}, {'measure': 'Overall safety of INXN-4001', 'timeFrame': '12 months', 'description': 'Incidence of cardiac-specific adverse events and study intervention-related serious adverse events'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['retrograde coronary sinus infusion', 'DNA', 'non-viral', 'triple effector plasmid', 'gene therapy', 'regenerative medicine', 'non-stem cell'], 'conditions': ['Heart Failure', 'Cardiovascular Diseases', 'Heart-Assist Device']}, 'descriptionModule': {'briefSummary': 'To evaluate the safety of retrograde coronary sinus infusion (RCSI) of a novel triple-effector plasmid (INXN-4001) in outpatient LVAD recipients as assessed by incidence of all study intervention-related adverse events occurring up to 6 months post-RCSI (primary endpoints), and to evaluate general safety by assessing incidence of cardiac specific adverse events and the incidence of related serious adverse events at intervals up to 12 months post-infusion (or until cardiac transplantation or death).', 'detailedDescription': 'This is a first-in-human, phase I, open label, safety study of INXN-4001 delivered via RCSI in patients with outpatient LVAD. Twelve stable patients with implanted LVAD for mechanical support of end stage heart failure have been allocated into 2 cohorts (6 subjects each) to evaluate the safety of infusing the same amount of INXN-4001 (80mg) in 2 volumes (40mL and 80mL) at 20mL/min rate. Ongoing safety assessments include clinical labs, physical exams, ECG and medical history collected during clinic visits at: pre-treatment, day 3, then 1, 3, 6, 9, and 12 months after dosing via RCSI. During visits, subjects complete the KCCQ questionnaire and are evaluated via 6-min walk test (6MWT) prior to and during an LVAD wean interval. Daily activity data is collected throughout the study using a wearable biosensor (Actigraph).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male and female adult patients with a stable LVAD implanted for end-stage heart failure\n* Must be managed in an outpatient setting and on stable medication regimen\n\nExclusion Criteria:\n\n* Women who are pregnant or nursing\n* Patients who have been on another clinical trial for heart failure in the last 90 days, or have received any stem cell or gene therapy within the previous year.\n* Patient is not able to complete a Six Minute Walk Test or unable to tolerate an LVAD wean in the past 3 months\n* Patient has an active infection requiring systemic antibiotics or an autoimmune disease requiring systemic immunosuppressants\n* Patient has a history of arrhythmia, uncontrolled diabetes, diabetic retinopathy, systemic lupus erythematosus, macular degeneration, hyper-coagulation, or stroke\n* Patient has had a myocardial infarction related to ischemia within the past 30 days\n* Patient has had certain prior surgeries such as organ transplant, cardiac transplantation, left ventricle reduction surgery, or cardiomyoplasty\n* Patient has infectious disease, such as hepatitis B or C, or human immunodeficiency virus (HIV)\n* Patient has a history of cancer within the past 3 years'}, 'identificationModule': {'nctId': 'NCT03409627', 'briefTitle': 'Novel INXN-4001 Triple Effector Plasmid in Heart Failure', 'organization': {'class': 'INDUSTRY', 'fullName': 'Triple-Gene, LLC'}, 'officialTitle': 'A Phase I, Open Label, Safety Study of INXN-4001 Delivered Via Retrograde Coronary Sinus Infusion in Patients With an Outpatient Left Ventricular Assist Device (LVAD)', 'orgStudyIdInfo': {'id': 'INXN-4001-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Group 1', 'description': 'Single infusion of INXN-4001, Dose 1', 'interventionNames': ['Biological: INXN-4001']}, {'type': 'EXPERIMENTAL', 'label': 'Group 2', 'description': 'Single infusion of INXN-4001, Dose 2', 'interventionNames': ['Biological: INXN-4001']}], 'interventions': [{'name': 'INXN-4001', 'type': 'BIOLOGICAL', 'description': 'Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001)', 'armGroupLabels': ['Group 1', 'Group 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85724', 'city': 'Tucson', 'state': 'Arizona', 'country': 'United States', 'facility': 'University of Arizona Sarver Heart Center', 'geoPoint': {'lat': 32.22174, 'lon': -110.92648}}, {'zip': '45219', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'The Lindner Research Center, The Christ Hospital Health Network', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}], 'overallOfficials': [{'name': 'David Bull, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Arizona'}, {'name': 'Ulrich Jorde, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Montefiore Medical Center'}, {'name': 'Gregory Egnaczyk, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'The Christ Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Triple-Gene, LLC', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Intrexon Corporation', 'class': 'INDUSTRY'}, {'name': 'Precigen, Inc', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}