NCT03409627

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1 heart-failure

Timeline
Completed

Started Apr 2018

Typical duration for phase_1 heart-failure

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 3, 2017

Completed
3 months until next milestone

First Posted

Study publicly available on registry

January 24, 2018

Completed
3 months until next milestone

Study Start

First participant enrolled

April 9, 2018

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 4, 2020

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 12, 2020

Completed
Last Updated

December 4, 2020

Status Verified

December 1, 2020

Enrollment Period

1.9 years

First QC Date

November 3, 2017

Last Update Submit

December 3, 2020

Conditions

Heart FailureCardiovascular DiseasesHeart-Assist Device

Keywords

retrograde coronary sinus infusionDNAnon-viraltriple effector plasmidgene therapyregenerative medicinenon-stem cell

Outcome Measures

Primary Outcomes (1)

  • 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

    6 months

Secondary Outcomes (4)

  • Wean tolerability following treatment with INXN-4001

    12 months

  • Quality of Life following treatment with INXN-4001

    12 months

  • Feasibility of biosensor activity tracking

    12 months

  • Overall safety of INXN-4001

    12 months

Study Arms (2)

Group 1

EXPERIMENTAL

Single infusion of INXN-4001, Dose 1

Biological: INXN-4001

Group 2

EXPERIMENTAL

Single infusion of INXN-4001, Dose 2

Biological: INXN-4001

Interventions

INXN-4001BIOLOGICAL

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

Group 1Group 2

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University of Arizona Sarver Heart Center

Tucson, Arizona, 85724, United States

Location

The Lindner Research Center, The Christ Hospital Health Network

Cincinnati, Ohio, 45219, United States

Location

MeSH Terms

Conditions

Heart FailureCardiovascular Diseases

Condition Hierarchy (Ancestors)

Heart Diseases

Study Officials

  • David Bull, MD

    University of Arizona

    PRINCIPAL INVESTIGATOR

  • Ulrich Jorde, MD

    Montefiore Medical Center

    PRINCIPAL INVESTIGATOR

  • Gregory Egnaczyk, MD

    The Christ Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 3, 2017

First Posted

January 24, 2018

Study Start

April 9, 2018

Primary Completion

March 4, 2020

Study Completion

August 12, 2020

Last Updated

December 4, 2020

Record last verified: 2020-12

Data Sharing

IPD Sharing
Will not share

Locations

US(2)