Novel INXN-4001 Triple Effector Plasmid in Heart Failure
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03409627 |
Recruitment Status :
Completed
First Posted : January 24, 2018
Last Update Posted : December 4, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Heart Failure Cardiovascular Diseases Heart-Assist Device | Biological: INXN-4001 | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 12 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | 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) |
Actual Study Start Date : | April 9, 2018 |
Actual Primary Completion Date : | March 4, 2020 |
Actual Study Completion Date : | August 12, 2020 |

Arm | Intervention/treatment |
---|---|
Experimental: Group 1
Single infusion of INXN-4001, Dose 1
|
Biological: INXN-4001
Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001) |
Experimental: Group 2
Single infusion of INXN-4001, Dose 2
|
Biological: INXN-4001
Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001) |
- To evaluate safety and feasibility of INXN-4001 [ Time Frame: 6 months ]As assessed by incidence of all study intervention-related adverse events occurring up to 6 months post- treatment intervention-related adverse events
- Wean tolerability following treatment with INXN-4001 [ Time Frame: 12 months ]Improvement in 6 minute walk duration and distance
- Quality of Life following treatment with INXN-4001 [ Time Frame: 12 months ]Change in Kansas City Cardiomyopathy Questionnaire responses
- Feasibility of biosensor activity tracking [ Time Frame: 12 months ]Ability to collect daily activity level as measured by wearable biosensor
- Overall safety of INXN-4001 [ Time Frame: 12 months ]Incidence of cardiac-specific adverse events and study intervention-related serious adverse events

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male and female adult patients with a stable LVAD implanted for end-stage heart failure
- Must be managed in an outpatient setting and on stable medication regimen
Exclusion Criteria:
- Women who are pregnant or nursing
- 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.
- Patient is not able to complete a Six Minute Walk Test or unable to tolerate an LVAD wean in the past 3 months
- Patient has an active infection requiring systemic antibiotics or an autoimmune disease requiring systemic immunosuppressants
- Patient has a history of arrhythmia, uncontrolled diabetes, diabetic retinopathy, systemic lupus erythematosus, macular degeneration, hyper-coagulation, or stroke
- Patient has had a myocardial infarction related to ischemia within the past 30 days
- Patient has had certain prior surgeries such as organ transplant, cardiac transplantation, left ventricle reduction surgery, or cardiomyoplasty
- Patient has infectious disease, such as hepatitis B or C, or human immunodeficiency virus (HIV)
- Patient has a history of cancer within the past 3 years

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03409627
United States, Arizona | |
University of Arizona Sarver Heart Center | |
Tucson, Arizona, United States, 85724 | |
United States, Ohio | |
The Lindner Research Center, The Christ Hospital Health Network | |
Cincinnati, Ohio, United States, 45219 |
Principal Investigator: | David Bull, MD | University of Arizona | |
Principal Investigator: | Ulrich Jorde, MD | Montefiore Medical Center | |
Principal Investigator: | Gregory Egnaczyk, MD | The Christ Hospital |
Responsible Party: | Triple-Gene, LLC |
ClinicalTrials.gov Identifier: | NCT03409627 |
Other Study ID Numbers: |
INXN-4001-001 |
First Posted: | January 24, 2018 Key Record Dates |
Last Update Posted: | December 4, 2020 |
Last Verified: | December 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
retrograde coronary sinus infusion DNA non-viral triple effector plasmid |
gene therapy regenerative medicine non-stem cell |
Heart Failure Cardiovascular Diseases Heart Diseases |