A Phase 1 interventional study of INXN-4001 in Heart Failure, Cardiovascular Diseases and Heart-Assist Device, sponsored by Triple-Gene, LLC. Completed at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-12-04.
Sponsored by Triple-Gene, LLC · Phase 1, Interventional, and Treatment
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).
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).
Exclusion Criteria:
Single infusion of INXN-4001, Dose 1
Biological: INXN-4001
Single infusion of INXN-4001, Dose 2
Biological: INXN-4001
Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001)
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
Time frame: 6 months
Wean tolerability following treatment with INXN-4001
Improvement in 6 minute walk duration and distance
Time frame: 12 months
Quality of Life following treatment with INXN-4001
Change in Kansas City Cardiomyopathy Questionnaire responses
Time frame: 12 months
Feasibility of biosensor activity tracking
Ability to collect daily activity level as measured by wearable biosensor
Time frame: 12 months
Overall safety of INXN-4001
Incidence of cardiac-specific adverse events and study intervention-related serious adverse events
Time frame: 12 months
Plan to share: No
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