Novel INXN-4001 Triple Effector Plasmid in Heart Failure

Heart Failure Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03409627
• Other study ID #: INXN-4001-001
• Status: Completed
• Phase: Phase 1
• Start date: April 9, 2018
• Est. completion date: August 12, 2020

Study information

• Verified date: December 2020
• Source: Triple-Gene, LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: August 12, 2020
• Est. primary completion date: March 4, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cardiovascular Diseases
• Heart Failure
• Heart-Assist Device

Intervention

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

Locations

Country	Name	City	State
United States	The Lindner Research Center, The Christ Hospital Health Network	Cincinnati	Ohio
United States	University of Arizona Sarver Heart Center	Tucson	Arizona

Sponsors (3)

Lead Sponsor	Collaborator
Triple-Gene, LLC	Intrexon Corporation, Precigen, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Wean tolerability following treatment with INXN-4001	Improvement in 6 minute walk duration and distance	12 months
Secondary	Quality of Life following treatment with INXN-4001	Change in Kansas City Cardiomyopathy Questionnaire responses	12 months
Secondary	Feasibility of biosensor activity tracking	Ability to collect daily activity level as measured by wearable biosensor	12 months
Secondary	Overall safety of INXN-4001	Incidence of cardiac-specific adverse events and study intervention-related serious adverse events	12 months