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Novel INXN-4001 Triple Effector Plasmid in Heart Failure

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ClinicalTrials.gov Identifier: NCT03409627
Recruitment Status : Completed
First Posted : January 24, 2018
Last Update Posted : December 4, 2020
Sponsor:
Collaborators:
Intrexon Corporation
Precigen, Inc
Information provided by (Responsible Party):
Triple-Gene, LLC

Tracking Information
First Submitted Date  ICMJE November 3, 2017
First Posted Date  ICMJE January 24, 2018
Last Update Posted Date December 4, 2020
Actual Study Start Date  ICMJE April 9, 2018
Actual Primary Completion Date March 4, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 30, 2018)		 To evaluate safety and feasibility of INXN-4001 [ Time Frame: 6 months ]
As assessed by incidence of all study intervention-related adverse events occurring up to 6 months post- treatment intervention-related adverse events
Original Primary Outcome Measures  ICMJE
 (submitted: January 17, 2018)		 To evaluate safety and feasibility at 6 months post INXN-4001 delivery [ Time Frame: 6 months ]
As assessed by incidence of all study intervention-related adverse events occurring up to 6 months post- treatment intervention-related adverse events
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 30, 2018)
  • Wean tolerability following treatment with INXN-4001 [ Time Frame: 12 months ]
    Improvement in 6 minute walk duration and distance
  • Quality of Life following treatment with INXN-4001 [ Time Frame: 12 months ]
    Change in Kansas City Cardiomyopathy Questionnaire responses
  • Feasibility of biosensor activity tracking [ Time Frame: 12 months ]
    Ability to collect daily activity level as measured by wearable biosensor
  • Overall safety of INXN-4001 [ Time Frame: 12 months ]
    Incidence of cardiac-specific adverse events and study intervention-related serious adverse events
Original Secondary Outcome Measures  ICMJE
 (submitted: January 17, 2018)		 Six-minute walk, KCCQ Questionaire [ Time Frame: 6 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Novel INXN-4001 Triple Effector Plasmid in Heart Failure
Official Title  ICMJE 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)
Brief 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).
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).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Heart Failure
  • Cardiovascular Diseases
  • Heart-Assist Device
Intervention  ICMJE Biological: INXN-4001
Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001)
Study Arms  ICMJE
  • Experimental: Group 1
    Single infusion of INXN-4001, Dose 1
    Intervention: Biological: INXN-4001
  • Experimental: Group 2
    Single infusion of INXN-4001, Dose 2
    Intervention: Biological: INXN-4001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 17, 2018)		 12
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE August 12, 2020
Actual Primary Completion Date March 4, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03409627
Other Study ID Numbers  ICMJE INXN-4001-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Triple-Gene, LLC
Study Sponsor  ICMJE Triple-Gene, LLC
Collaborators  ICMJE
  • Intrexon Corporation
  • Precigen, Inc
Investigators  ICMJE
Principal Investigator: David Bull, MD University of Arizona
Principal Investigator: Ulrich Jorde, MD Montefiore Medical Center
Principal Investigator: Gregory Egnaczyk, MD The Christ Hospital
PRS Account Triple-Gene, LLC
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP