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Novel INXN-4001 Triple Effector Plasmid in Heart Failure

Primary Purpose

Heart Failure, Cardiovascular Diseases, Heart-Assist Device

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

INXN-4001

About this trial

This is an interventional treatment trial for Heart Failure focused on measuring retrograde coronary sinus infusion, DNA, non-viral, triple effector plasmid, gene therapy, regenerative medicine, non-stem cell

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

University of Arizona Sarver Heart Center
The Lindner Research Center, The Christ Hospital Health Network

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Group 1

Group 2

Arm Description

Single infusion of INXN-4001, Dose 1

Single infusion of INXN-4001, Dose 2

Outcomes

Primary Outcome Measures

To evaluate safety and feasibility of INXN-4001

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

Wean tolerability following treatment with INXN-4001

Improvement in 6 minute walk duration and distance

Quality of Life following treatment with INXN-4001

Change in Kansas City Cardiomyopathy Questionnaire responses

Feasibility of biosensor activity tracking

Ability to collect daily activity level as measured by wearable biosensor

Overall safety of INXN-4001

Incidence of cardiac-specific adverse events and study intervention-related serious adverse events

Full Information

NCT ID

NCT03409627

First Posted

November 3, 2017

Last Updated

December 3, 2020

Sponsor

Triple-Gene, LLC

Collaborators

Intrexon Corporation, Precigen, Inc

1. Study Identification

Unique Protocol Identification Number

NCT03409627

Brief Title

Novel INXN-4001 Triple Effector Plasmid in Heart Failure

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)

Study Type

Interventional

2. Study Status

Record Verification Date

December 2020

Overall Recruitment Status

Completed

Study Start Date

April 9, 2018 (Actual)

Primary Completion Date

March 4, 2020 (Actual)

Study Completion Date

August 12, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Triple-Gene, LLC

Collaborators

Intrexon Corporation, Precigen, Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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).

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Heart Failure, Cardiovascular Diseases, Heart-Assist Device

Keywords

retrograde coronary sinus infusion, DNA, non-viral, triple effector plasmid, gene therapy, regenerative medicine, non-stem cell

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Group 1

Arm Type

Experimental

Arm Description

Single infusion of INXN-4001, Dose 1

Arm Title

Group 2

Arm Type

Experimental

Arm Description

Single infusion of INXN-4001, Dose 2

Intervention Type

Biological

Intervention Name(s)

INXN-4001

Intervention Description

Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001)

Primary Outcome Measure Information:

Title

To evaluate safety and feasibility of INXN-4001

Description

As assessed by incidence of all study intervention-related adverse events occurring up to 6 months post- treatment intervention-related adverse events

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Wean tolerability following treatment with INXN-4001

Description

Improvement in 6 minute walk duration and distance

Time Frame

12 months

Title

Quality of Life following treatment with INXN-4001

Description

Change in Kansas City Cardiomyopathy Questionnaire responses

Time Frame

12 months

Title

Feasibility of biosensor activity tracking

Description

Ability to collect daily activity level as measured by wearable biosensor

Time Frame

12 months

Title

Overall safety of INXN-4001

Description

Incidence of cardiac-specific adverse events and study intervention-related serious adverse events

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David Bull, MD

Organizational Affiliation

University of Arizona

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Ulrich Jorde, MD

Organizational Affiliation

Montefiore Medical Center

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Gregory Egnaczyk, MD

Organizational Affiliation

The Christ Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Arizona Sarver Heart Center

City

Tucson

State/Province

Arizona

ZIP/Postal Code

85724

Country

United States

Facility Name

The Lindner Research Center, The Christ Hospital Health Network

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45219

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Novel INXN-4001 Triple Effector Plasmid in Heart Failure

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