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Purpose

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).

Conditions

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Group 1 		Single infusion of INXN-4001, Dose 1
  • Biological: INXN-4001
    Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001)
Experimental
Group 2 		Single infusion of INXN-4001, Dose 2
  • Biological: INXN-4001
    Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001)

More Details

Status
Completed
Sponsor
Triple-Gene, LLC

Study Contact

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).

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.