Novel INXN-4001 Triple Effector Plasmid in Heart Failure

Triple-Gene, LLC

Status and phase

Completed

Phase 1

Conditions

Heart-Assist Device

Cardiovascular Diseases

Heart Failure

Treatments

Biological: INXN-4001

Study type

Interventional

Funder types

Industry

Identifiers

NCT03409627

INXN-4001-001

Details and patient eligibility

About

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).

Full 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).

Enrollment

12 patients

Sex

All

Ages

18+ years old

Volunteers

No 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