Novel INXN-4001 Triple Effector Plasmid in Heart Failure

NCT ID: NCT03409627

Last Updated: 2020-12-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-04-09
• Study Completion Date: 2020-08-12

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).

Conditions

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Group 1

Single infusion of INXN-4001, Dose 1

Group Type: EXPERIMENTAL

INXN-4001

Intervention Type: BIOLOGICAL

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

Group 2

Single infusion of INXN-4001, Dose 2

Group Type: EXPERIMENTAL

INXN-4001

Intervention Type: BIOLOGICAL

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

Interventions

INXN-4001

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

Intervention Type: BIOLOGICAL

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Intrexon Corporation

INDUSTRY

Sponsor Role: collaborator

Precigen, Inc

INDUSTRY

Sponsor Role: collaborator

Triple-Gene, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

David Bull, MD

Role: PRINCIPAL_INVESTIGATOR

University of Arizona

Ulrich Jorde, MD

Role: PRINCIPAL_INVESTIGATOR

Montefiore Medical Center

Gregory Egnaczyk, MD

Role: PRINCIPAL_INVESTIGATOR

The Christ Hospital

Locations

University of Arizona Sarver Heart Center

Tucson, Arizona, United States

The Lindner Research Center, The Christ Hospital Health Network

Cincinnati, Ohio, United States

Countries

United States

Other Identifiers

INXN-4001-001

Identifier Type: -

Identifier Source: org_study_id