Congestive Heart FailurePhase 1NAN-CS101NCT04179643
AB-1002 in Patients With Class III Heart Failure
A Phase 1 study testing 3 x 10e13vg AB-1002 in people with Congestive Heart Failure and Heart Failure, run by AskBio Inc at 4 sites in United States.
Trial summary
This is a Phase 1, prospective, multi-center, open-label, sequential dose escalation study to explore the safety, feasibility, and efficacy of a single intracoronary infusion of AB-1002 in patients with NYHA Class III heart failure. 11 participants with non-ischemic cardiomyopathy were enrolled. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Congestive Heart Failure, Heart Failure, Heart Disease, Ischemic
- What is being tested?
- 3 x 10e13vg AB-1002
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 4 sitesUnited States
- Study dates
- 20 Nov 2019to 23 Jul 2026
- 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
- Age >18 years of age
- Chronic non-ischemic cardiomyopathy
- LVEF 15% ≤ 30% by transthoracic echocardiography (TTE) within 6 months prior to enrollment
- NYHA Class III HF for a minimum of 3 months HF despite appropriate medical therapy (defined below):
- Treatment with appropriate HF therapy as tolerated, including, but not limited to:
- Beta blocker therapy and angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) or sacubitril/valsartan combination therapy (Entresto) for ≥ 90 days prior to enrollment. May also receive aldosterone antagonist therapy. Doses of the above medications must be stable for ≥ 30 days prior to enrollment; and
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- Cardiac resynchronization therapy (CRT), if clinically indicated, must have been implanted ≥ 90 days prior to enrollment. Internal cardioverter defibrillator (ICD) must be implanted, if clinically indicated ≥ 30 days prior to enrollment
- Females of childbearing potential must use at least one of the following acceptable birth control methods throughout the study and for 6 months after IP administration:
- Surgically sterile (bilateral tubal ligation, hysterectomy, bilateral oophorectomy) 6 months minimum prior to IP administration
- Intrauterine device in place for at least 90 days prior to receiving IP
- Barrier methods (diaphragm plus spermicide or condom) starting at least 30 days prior to receiving IP
- Abstinence (the subject must be willing to remain abstinent from screening to 6 months after receiving IP). Females are allowed to claim abstinence as their method of contraception only when it is the preferred and usual lifestyle of the subject
- Surgical sterilization of the partner(s) (vasectomy) for >180 days prior to IP administration
- Hormonal contraceptives starting > 90 days prior to IP. If hormonal contraceptives are started less than 90 days prior to receiving IP, subjects must agree to use a barrier method (diaphragm plus spermicide or condom) from screening through 90 days after initiation of hormonal contraceptives
- Males subjects capable of fathering a child:
- Must agree to use a condom from IP administration through 6 months after the time of IP administration
- Must agree not to donate sperm for 6 months after time of receiving IP
- Documented evidence of vasectomy in males for 180 days minimum prior to receiving IP is an acceptable form of contraception
- Males who claim abstinence as their method of contraception are allowed provided they agree to use barrier methods should they become sexually active from screening through 6 months after receiving IP. Males are allowed to claim abstinence as their method of contraception only when it is the preferred and usual lifestyle of the subject
- Ability to sign Informed Consent Form (ICF) and Release of Medical Information Form
- Appropriate candidate for protocol-specified intracoronary infusion in the judgment of the infusing interventional cardiologist
- Cohort 3: medical history documentation of PLN-R14Del mutation and an ICD in situ (at least 30 days prior to enrollment)
Who cannot join
- Chronic ischemic cardiomyopathy
- Intravenous (IV) inotropic therapy, intra-aortic balloon pump (IABP) or percutaneous cardiac assist device therapy within 30 days prior to enrollment
- Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm
- Cardiac surgery or percutaneous coronary intervention (PCI) within 30 days prior to enrollment
- Third degree heart block
- Clinically significant myocardial infarction (MI) in the judgment of the subject's physician (e.g., ST elevation MI [STEMI] or large non-STEMI) within 6 months prior to enrollment
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- Prior heart transplantation, left ventricular reduction surgery (LVRS), cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device), surgically implanted LVAD or cardiac shunt
- Likely to receive cardiac resynchronization therapy, cardiomyoplasty, LV reduction surgery, heart transplant, conventional revascularization procedure, or valvular repair within 3 months of IP dosing
- Known hypersensitivity to contrast dyes used for angiography; history of, or likely need for, high-dose steroid pretreatment prior to contrast angiography
- Expected survival < 1 year in the judgment of the investigator
- Active or suspected infection within 48 hours prior to enrollment as evidenced by fever or positive culture
- Known intrinsic liver disease (e.g., cirrhosis, hepatitis A, chronic hepatitis B or hepatitis C virus infection). If serology is positive and PCR is negative, subject may be eligible (confirm with medical monitor).
- Liver function tests (alanine aminotransferase [ALT], aspartate aminotransferase [AST], alkaline phosphatase) > 2x upper limit of normal (ULN) within 30 days prior to enrollment.
- Renal Failure, dialysis dependent or serum creatinine > 2.5 mg/dl within 30 days prior to enrollment
- Bleeding diathesis or thrombocytopenia defined as platelets <50,000 platelets/μL within 30 days prior to enrollment
- Anemia defined as hemoglobin <10 g/dL or transfusion dependent within 30 days prior to enrollment
- Neutropenia defined as absolute neutrophils <1500 mm3 within 30 days prior to enrollment
- Known AIDS or HIV-positive status, or a previous diagnosis of immunodeficiency with an absolute neutrophil count <1000 cells/mm3
- Previous participation in a study of gene transfer
- Receiving investigational intervention or participating in another clinical study within 30 days or within 5 half-lives of another investigational drug administration prior to administration of NAN-101 that may impact the therapeutic potential of NAN-101.
- Pregnancy or breastfeeding at the time of screening
Trial locations and contacts
4 sites in 1 country.
United States4 sites
The Linder Center for Education and Research at The Christ Hospital
Cincinnati, Ohio, 45219
The Ohio State University
Columbus, Ohio, 43210
University of Wisconsin at Madison
Madison, Wisconsin, 53792
Minneapolis Heart Foundation Institute
Minneapolis, Minnesota, 55407
About this study
Official title: Phase 1 Open Label, Dose Escalation Trial of Intracoronary Infusion of AB-1002 in Subjects With NYHA Class III Heart Failure
This is a Phase 1, prospective, multi-center, open-label, sequential dose escalation study to explore the safety, feasibility, and efficacy of a single intracoronary infusion of AB-1002 in patients with NYHA Class III heart failure. 11 participants with non-ischemic cardiomyopathy were enrolled. All participants were followed until 12 months post treatment intervention, and then underwent long-term follow-up via semi-structured telephone questionnaires every 6 months for an additional 24 months (+/- 30 days).
Read the detailed description
The safety endpoints will be assessed over the 12-month follow-up period
- Adverse Events
- Clinical laboratory tests
- Vital signs
- Electrocardiograms
Efficacy Endpoints
The efficacy endpoints assessed from baseline to 6 and 12 months will include the following:
- Observed and changes from baseline in peak VO2 assessed by cardiopulmonary exercise testing
- Observed and changes from baseline in 6-minute walk test
- Observed and changes from baseline in NYHA Classification
- Total number of days alive out-of-hospital
The secondary efficacy endpoints will explore efficacy. Functional endpoints will be assessed as changes from baseline to 6 and 12 months following investigational product administration as indicated. These endpoints include:
Functional Status \& Hospitalizations
- Peak VO2 assessed by cardiopulmonary exercise testing
- 6-minute walk test
- New York Heart Association (NYHA) Classification
- Total number of days alive out-of-hospital (as well as total days out-of-hospital as a % of total days alive post study intervention)
Physiologic Assessments at 6 and 12 months compared to baseline
- Echocardiographic assessments of LVEF, LVEVD, LVEDVI, VLESV, LVEVI, SpI and GLSand degree of mitral regurgitation
- NT-proBNP level
Quality of Life at Week 8, 6 and 12 months compared to baseline
- Health related quality of life as assessed by Minnesota Living with Heart Failure Questionnaire (MLWHFQ) and Kansas City Cardiomyopathy Questionnaire
The following endpoints will also be measured over the 12 month follow-up period and long-term follow-up period (until month 36 post-intervention):
- Survival
- Cardiac transplantation
- Left ventricular assist device (LVAD) implantation
- Conditions
- Congestive Heart Failure, Heart Failure, Heart Disease, Ischemic, Cardiovascular Diseases, Heart Failure, Systolic, Heart Failure，Congestive
- Being tested
- 3 x 10e13vg AB-1002
- Medical subject headings
- Heart Failure, Myocardial Ischemia, Cardiovascular Diseases, Heart Failure, Systolic, Arrhythmias, Cardiac, Heart Failure, Diastolic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3 x 10e13vg AB-1002Biological
There are 2 components to AB-1002. The first is an active I-1 transgene (AA 1-65 with T35D), and the second is the vector, BNP116, which delivers the gene selectively to the heart after intracoronary administration.
| Study group | Role | What they receive |
|---|---|---|
| 3.25E13vg AB-1002 | Experimental | Intracoronary Infusion of 3.25E13vg AB-1002 up to 6 subjects Receives: Biological: 3 x 10e13vg AB-1002 |
| 1.08E14vg AB-1002 | Experimental | Intracoronary Infusion of 1.08E14vg AB-1002 to 6 subjects Receives: Biological: 3 x 10e13vg AB-1002 |
| PLN-R14Del patients: 3.25E13vg AB-1002 | Experimental | Intracoronary Infusion of AB-1002 at 3.25E13vg up to 6 subjects with PLN-R14Del genetic mutation Receives: Biological: 3 x 10e13vg AB-1002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Observed and change from baseline in Peak VO2
Cardiopulmonary exercise testing using a modified Bruce protocol
Measured: Measured at screening, month 6, 9 and month 12
Observed and change from baseline in Echocardiographic assessment in Left Ventricular Ejection Fraction
Echocardiography LVEF measurement
Measured: Measured at screening, 18-24 hours post intervention, week 4, Month 3, Month 6 and Month 12
Secondary outcomes (1)
Observed and change from baseline in 6-minute walk test distance
Analysis of Percent predicted in heart failure subjects compared to normal subjects
Measured: Measured at screening, Month 3, Month 6 and month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2019
- Study start20 Nov 2019
- Primary completion20 Aug 2024
- Study completion23 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congestive 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- AskBio Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04179643
- Sponsor's protocol ID
- NAN-CS101
- Acronym
- NAN-CS101
- Status last verified
- Aug 2026
- First submitted
- 6 Sep 2019
- Last updated
- 6 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Henry TD, Chung ES, Alvisi M, Sethna F, Murray DR, Traverse JH, Roessig L, Roberts L, Reddy S, Chen Y, Ozkan TG, Webb S, Mittal M, Ervin L, Sadek H, Mikhail S, Haghighi K, Jiang C, Samulski RJ, Kranias EG, Tretiakova AP, Hajjar RJ. Cardiotropic AAV gene therapy for heart failure: a phase 1 trial. Nat Med. 2025 Nov;31(11):3845-3852. doi: 10.1038/s41591-025-04011-z. Epub 2025 Oct 21.
PubMed 41120766
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Open Label, Dose Escalation Trial of Intracoronary Infusion of AB-1002 in Subjects With NYHA Class III Heart Failure
- ClinicalTrials.gov ID
- NCT04179643
- Acronym / sponsor ID
- NAN-CS101 / NAN-CS101
- Conditions
- Congestive Heart Failure, Heart Failure, Heart Disease, Ischemic, Cardiovascular Diseases, Heart Failure, Systolic, Heart Failure，Congestive
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- AskBio IncIndustry (pharma, biotech, devices)
- Study start
- 20 Nov 2019 (actual)
- Primary completion
- 20 Aug 2024 (actual)
- Study completion
- 23 Jul 2026 (actual)
- First posted
- 27 Nov 2019 (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.
This is a Phase 1, prospective, multi-center, open-label, sequential dose escalation study to explore the safety, feasibility, and efficacy of a single intracoronary infusion of AB-1002 in patients with NYHA Class III heart failure. 11 participants with non-ischemic cardiomyopathy were enrolled. All participants were followed until 12 months post treatment intervention, and then underwent long-term follow-up via semi-structured telephone questionnaires every 6 months for an additional 24 months (+/- 30 days).
Detailed description
The safety endpoints will be assessed over the 12-month follow-up period
- Adverse Events
- Clinical laboratory tests
- Vital signs
- Electrocardiograms
Efficacy Endpoints
The efficacy endpoints assessed from baseline to 6 and 12 months will include the following:
- Observed and changes from baseline in peak VO2 assessed by cardiopulmonary exercise testing
- Observed and changes from baseline in 6-minute walk test
- Observed and changes from baseline in NYHA Classification
- Total number of days alive out-of-hospital
The secondary efficacy endpoints will explore efficacy. Functional endpoints will be assessed as changes from baseline to 6 and 12 months following investigational product administration as indicated. These endpoints include:
Functional Status \& Hospitalizations
- Peak VO2 assessed by cardiopulmonary exercise testing
- 6-minute walk test
- New York Heart Association (NYHA) Classification
- Total number of days alive out-of-hospital (as well as total days out-of-hospital as a % of total days alive post study intervention)
Physiologic Assessments at 6 and 12 months compared to baseline
- Echocardiographic assessments of LVEF, LVEVD, LVEDVI, VLESV, LVEVI, SpI and GLSand degree of mitral regurgitation
- NT-proBNP level
Quality of Life at Week 8, 6 and 12 months compared to baseline
- Health related quality of life as assessed by Minnesota Living with Heart Failure Questionnaire (MLWHFQ) and Kansas City Cardiomyopathy Questionnaire
The following endpoints will also be measured over the 12 month follow-up period and long-term follow-up period (until month 36 post-intervention):
- Survival
- Cardiac transplantation
- Left ventricular assist device (LVAD) implantation
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 3.25E13vg AB-1002 | Experimental | Biological: 3 x 10e13vg AB-1002Intracoronary Infusion of 3.25E13vg AB-1002 up to 6 subjects |
| 1.08E14vg AB-1002 | Experimental | Biological: 3 x 10e13vg AB-1002Intracoronary Infusion of 1.08E14vg AB-1002 to 6 subjects |
| PLN-R14Del patients: 3.25E13vg AB-1002 | Experimental | Biological: 3 x 10e13vg AB-1002Intracoronary Infusion of AB-1002 at 3.25E13vg up to 6 subjects with PLN-R14Del genetic mutation |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Age >18 years of age
- Chronic non-ischemic cardiomyopathy
- LVEF 15% ≤ 30% by transthoracic echocardiography (TTE) within 6 months prior to enrollment
- NYHA Class III HF for a minimum of 3 months HF despite appropriate medical therapy (defined below):
- Females of childbearing potential must use at least one of the following acceptable birth control methods throughout the study and for 6 months after IP administration:
Key exclusion criteria (21 listed)
- Chronic ischemic cardiomyopathy
- Intravenous (IV) inotropic therapy, intra-aortic balloon pump (IABP) or percutaneous cardiac assist device therapy within 30 days prior to enrollment
- Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm
- Cardiac surgery or percutaneous coronary intervention (PCI) within 30 days prior to enrollment
- Third degree heart block
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 |
|---|---|---|
| 3 x 10e13vg AB-1002There are 2 components to AB-1002. The first is an active I-1 transgene (AA 1-65 with T35D), and the second is the vector, BNP116, which delivers the gene selectively to the heart after intracoronary administration. | Biological | 1.08E14vg AB-1002, 3.25E13vg AB-1002, PLN-R14Del patients: 3.25E13vg AB-1002 |
Primary outcome
Observed and change from baseline in Peak VO2
Time frame: Measured at screening, month 6, 9 and month 12
Cardiopulmonary exercise testing using a modified Bruce protocol
Observed and change from baseline in Echocardiographic assessment in Left Ventricular Ejection Fraction
Time frame: Measured at screening, 18-24 hours post intervention, week 4, Month 3, Month 6 and Month 12
Echocardiography LVEF measurement
1 secondary outcome
Observed and change from baseline in 6-minute walk test distance
Time frame: Measured at screening, Month 3, Month 6 and month 12
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 6 Aug 2026.
- Derived from this study: PubMed 41120766Henry TD, Chung ES, Alvisi M, Sethna F, Murray DR, Traverse JH, Roessig L, Roberts L, Reddy S, Chen Y, Ozkan TG, Webb S, Mittal M, Ervin L, Sadek H, Mikhail S, Haghighi K, Jiang C, Samulski RJ, Kranias EG, Tretiakova AP, Hajjar RJ. Cardiotropic AAV gene therapy for heart failure: a phase 1 trial. Nat Med. 2025 Nov;31(11):3845-3852. doi: 10.1038/s41591-025-04011-z. Epub 2025 Oct 21.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
4 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 3 x 10e13vg AB-1002 in people with Congestive Heart Failure and Heart Failure, run by AskBio Inc at 4 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 22 inclusion and 21 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 6 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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?
Observed and change from baseline in Peak VO2 (time frame: Measured at screening, month 6, 9 and month 12). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04179643, which also lists 1 linked publication.
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 6 Aug 2026; AzTrials last refreshed its copy on 6 Nov 2026.