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Novel INXN-4001 Triple Effector Plasmid in Heart Failure

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. Identifier: NCT03409627
Recruitment Status : Completed
First Posted : January 24, 2018
Last Update Posted : December 4, 2020
Intrexon Corporation
Precigen, Inc
Information provided by (Responsible Party):
Triple-Gene, LLC

Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Heart Failure Cardiovascular Diseases Heart-Assist Device Biological: INXN-4001 Phase 1

Detailed Description:
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).

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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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Failure

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)

Primary Outcome Measures :
  1. 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

Secondary Outcome Measures :
  1. Wean tolerability following treatment with INXN-4001 [ Time Frame: 12 months ]
    Improvement in 6 minute walk duration and distance

  2. Quality of Life following treatment with INXN-4001 [ Time Frame: 12 months ]
    Change in Kansas City Cardiomyopathy Questionnaire responses

  3. Feasibility of biosensor activity tracking [ Time Frame: 12 months ]
    Ability to collect daily activity level as measured by wearable biosensor

  4. Overall safety of INXN-4001 [ Time Frame: 12 months ]
    Incidence of cardiac-specific adverse events and study intervention-related serious adverse events

Information from the National Library of Medicine

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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

Information from the National Library of Medicine

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 identifier (NCT number): NCT03409627

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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
Sponsors and Collaborators
Triple-Gene, LLC
Intrexon Corporation
Precigen, Inc
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Principal Investigator: David Bull, MD University of Arizona
Principal Investigator: Ulrich Jorde, MD Montefiore Medical Center
Principal Investigator: Gregory Egnaczyk, MD The Christ Hospital
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Responsible Party: Triple-Gene, LLC 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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Triple-Gene, LLC:
retrograde coronary sinus infusion
triple effector plasmid
gene therapy
regenerative medicine
non-stem cell
Additional relevant MeSH terms:
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Heart Failure
Cardiovascular Diseases
Heart Diseases