Novel INXN-4001 Triple Effector Plasmid in Heart Failure

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).

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