Heart FailurePhase 1NCT03409627
Novel INXN-4001 Triple Effector Plasmid in Heart Failure
A Phase 1 study testing INXN-4001 in people with Heart Failure and Cardiovascular Diseases, run by Triple-Gene, LLC at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Heart Failure, Cardiovascular Diseases, Heart-Assist Device
- What is being tested?
- INXN-4001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- 9 Apr 2018to 12 Aug 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
The Lindner Research Center, The Christ Hospital Health Network
Cincinnati, Ohio, 45219
University of Arizona Sarver Heart Center
Tucson, Arizona, 85724
About this study
Official title: 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)
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).
Read the 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).
- Conditions
- Heart Failure, Cardiovascular Diseases, Heart-Assist Device
- Being tested
- INXN-4001
- Keywords
- retrograde coronary sinus infusion, DNA, non-viral, triple effector plasmid, gene therapy, regenerative medicine, non-stem cell
- Medical subject headings
- Heart Failure, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INXN-4001Biological
Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001)
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Single infusion of INXN-4001, Dose 1 Receives: Biological: INXN-4001 |
| Group 2 | Experimental | Single infusion of INXN-4001, Dose 2 Receives: Biological: INXN-4001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 6 months
Secondary outcomes (4)
Wean tolerability following treatment with INXN-4001
Improvement in 6 minute walk duration and distance
Measured: 12 months
Quality of Life following treatment with INXN-4001
Change in Kansas City Cardiomyopathy Questionnaire responses
Measured: 12 months
Feasibility of biosensor activity tracking
Ability to collect daily activity level as measured by wearable biosensor
Measured: 12 months
Overall safety of INXN-4001
Incidence of cardiac-specific adverse events and study intervention-related serious adverse events
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2018
- Study start9 Apr 2018
- Primary completion4 Mar 2020
- Study completion12 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heart FailureStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Triple-Gene, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Intrexon Corporation, Precigen, Inc
- Responsible party
- Sponsor
- Investigators
- David Bull, MD, Principal investigator, University of ArizonaUlrich Jorde, MD, Principal investigator, Montefiore Medical CenterGregory Egnaczyk, MD, Principal investigator, The Christ Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03409627
- Sponsor's protocol ID
- INXN-4001-001
- Status last verified
- Dec 2020
- First submitted
- 3 Nov 2017
- Last updated
- 4 Dec 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT03409627
- Acronym / sponsor ID
- INXN-4001-001
- Conditions
- Heart Failure, Cardiovascular Diseases, Heart-Assist Device
- Keywords
- retrograde coronary sinus infusion, DNA, non-viral, triple effector plasmid, gene therapy, regenerative medicine, non-stem cell
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Triple-Gene, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Intrexon Corporation, Precigen, Inc
- Study start
- 9 Apr 2018 (actual)
- Primary completion
- 4 Mar 2020 (actual)
- Study completion
- 12 Aug 2020 (actual)
- First posted
- 24 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Biological: INXN-4001Single infusion of INXN-4001, Dose 1 |
| Group 2 | Experimental | Biological: INXN-4001Single infusion of INXN-4001, Dose 2 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| INXN-4001Retrograde coronary sinus infusion of triple effector plasmid (INXN-4001) | Biological | Group 1, Group 2 |
Primary outcome
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
4 secondary outcomes
Wean tolerability following treatment with INXN-4001
Time frame: 12 months
Quality of Life following treatment with INXN-4001
Time frame: 12 months
Feasibility of biosensor activity tracking
Time frame: 12 months
Overall safety of INXN-4001
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Bull, MD principal investigator
University of Arizona
Ulrich Jorde, MD principal investigator
Montefiore Medical Center
Gregory Egnaczyk, MD principal investigator
The Christ Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing INXN-4001 in people with Heart Failure and Cardiovascular Diseases, run by Triple-Gene, LLC at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate safety and feasibility of INXN-4001 (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2020.