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Congestive Heart FailurePhase 2GenePHITNCT05598333

Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure

A Phase 2 study testing AB-1002 in people with Congestive Heart Failure, run by AskBio Inc at 96 sites in 12 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial study to evaluate the safety and efficacy of a single dose of AB-1002, administered via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and NYHA Class III symptoms… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Congestive Heart Failure
What is being tested?
AB-1002
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
150 participants
Where
96 sitesin 12 countries
Study dates
20 Oct 2023to 31 Dec 2030
Phase
Phase 2
Are there results?
Not yet: the study is under way

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

  • Subject must be age ≥18 years of age, at the time of signing the informed consent.
  • Chronic non-ischemic cardiomyopathy
  • 15% ≤ LVEF ≤ 35% by transthoracic echocardiography (TTE) at screening
  • 6MWT >50 meters
  • Medically stable, NYHA Class III HF for a minimum of 4 weeks while on appropriate medical therapy (defined below) including, but not limited to:
  • Beta blocker therapy and ACE inhibitor or angiotensin receptor blocker (ARB) or sacubitril/valsartan combination therapy (Entresto) for ≥ 90 days prior to enrollment.
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  • May also receive aldosterone antagonist therapy. Doses of the above medications must be stable for ≥ 30 days prior to enrollment; and
  • Cardiac resynchronization therapy (Zareba et al 2011), 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.
  • Women 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 and should also be advised of the benefit for a female partner to use a highly effective method of contraception as a condom may break or leak when having sexual intercourse with a woman of childbearing potential who is not currently pregnant 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
  • Appropriate candidate for protocol-specified intracoronary infusion in the judgment of the infusing interventional cardiologist

Who cannot join

  • Subjects are excluded from the study if any of the following criteria apply:
  • Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease
  • 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 screening
  • Uncorrected Third degree heart block
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  • 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
  • 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 in judgement of investigator.
  • Known hypersensitivity to contrast dyes (not easily controlled by antihistamines) 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 intra-coronary infusion 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 known to be 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.
  • Chronic Kidney Disease Stage 5, dialysis dependent or eGFR<15 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 of another investigational drug administration prior to administration of AB-1002 that may impact the therapeutic potential of AB-1002.
  • Pregnancy or breastfeeding or plans to become pregnant within the next 12 months at the time of screening
  • Subjects with any other condition which in the opinion of the investigator would preclude participation in the study (including risk for non-compliance and any intercurrent conditions that pose an undue medical hazard, or which could interfere with the interpretation of the study results)
  • Malignant neoplasm within 5 years of dosing, with the exception of those with negligible risk of metastasis or death (such as adequately treated carcinoma in situs of the cervix, basal or squamous cell skin cancer, localized prostate cancer or ductal carcinoma in situ)
  • Any documented history of non-compliance with medications, illicit drug use or laboratory evidence of illicit drug use during screen period

Trial locations and contacts

96 sites in 12 countries.

96 sites in 12 countries

United States37 sites

  • LVH Cardiology

    Allentown, Pennsylvania, 18103

  • Augusta University

    Augusta, Georgia, 30912

  • Cardiology P.C. Birmingham

    Birmingham, Alabama, 35211

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • Medical University of South Carolina (MUSC) Medical Center

    Charleston, South Carolina, 29425

  • The Christ Hospital / The Linder Center for Research

    Cincinnati, Ohio, 45219

  • University of Cincinnati

    Cincinnati, Ohio, 45219

  • Baycare Medical Group

    Clearwater, Florida, 33756

  • MetroHealth Medical Center

    Cleveland, Ohio, 44109

  • The Ohio State University

    Columbus, Ohio, 43210

  • University of Miami

    Coral Gables, Florida, 33146

  • Baylor Scott & White Advanced Heart Failure Clinic - Dallas

    Dallas, Texas, 75246

  • Duke University

    Durham, North Carolina, 27705

  • University of Florida

    Gainesville, Florida, 32610

  • Stern Cardiovascular

    Germantown, Tennessee, 38138

  • Baylor College of Medicine (BCM) - Baylor Heart Clinic

    Houston, Texas, 77030

  • Houston Methodist Debakey Cardiology Associates

    Houston, Texas, 77030

  • University of Iowa

    Iowa City, Iowa, 52242

  • University of California Irvine Medical Center

    Irvine, California, 92697

  • University of Kansas Medical Center (KUMC)

    Kansas City, Kansas, 66160

  • Aurora Saint Luke's Health System

    Kansas City, Missouri, 64111

  • University of California San Diego

    La Jolla, California, 92093

  • University of Wisconsin

    Madison, Wisconsin, 53792

  • Minneapolis Heart Institute Foundation

    Minneapolis, Minnesota, 55407

  • Morristown Medical Center - Cardiology

    Morristown, New Jersey, 07962

  • Mt. Sinai New York

    New York, New York, 10029

  • Loyola Medicine Burr Ridge

    Oakbrook Terrace, Illinois, 60181

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

  • Renown Health

    Reno, Nevada, 89502

  • Mayo Clinic - Minnesota

    Rochester, Minnesota, 55905

  • University of California San Francisco

    San Francisco, California, 94143

  • University of Washington

    Seattle, Washington, 98195

  • St. Louis University

    St Louis, Missouri, 63117

  • Stony Brook

    Stony Brook, New York, 11794

  • Montefiore Medical Center

    The Bronx, New York, 10461

  • University of Arizona Sarvor Heart Center

    Tucson, Arizona, 85724

Other trials in United States

Canada10 sites

  • CIUSSS Saguenay Lac-St-Jean

    Chicoutimi, Quebec, G7H5H6

  • London Health Sciences Centre

    London, Ontario, N6A5A5

  • Institut de Cardiologie de Montréal

    Montreal, Quebec, H1T1C8

  • Centre Hospitalier de l'Université de Montréal - CHUM

    Montreal, Quebec, H2X3E4

  • Jewish General Hospital

    Montreal, Quebec, H3T1E2

  • CHU de Québec - Université Laval

    Québec, Quebec, G1R2J6

  • Institut Universitaire de Cardiologie et de Pneumologie de Québec - IUCPQ

    Québec, Quebec, G1V4G5

  • Memorial University Hospital

    St. John's, Newfoundland and Labrador, A1B3V6

  • St. Michael's Hospital

    Toronto, Ontario, M5B1W8

  • St. Boniface Hospital

    Winnipeg, Manitoba, R2H2A6

Other trials in Canada

Bulgaria8 sites

  • Multiprofile Hospital for Active Treatment Sveti Georgi OOD

    Pernik, 2300

  • Specialized Hospital For Active Cardiology Treatment Medica Kor EAD

    Rousse, 7013

  • Multiprofile Hospital for Active Treatment "South West Hospital" OOD

    Sandanski, 2802

  • Multiprofile Hospital for Active Treatment 'National Cardiology Hospital' EAD

    Sofia, 1309

  • Acibadem City Clinic University Multiprofile Hospital For Active Treatment Tokuda EAD

    Sofia, 1407

  • University Multiprofile Hospital for Active Treatment 'Sveta Ekaterina' EAD

    Sofia, 1431

  • University Multiprofile Hospital for Active Treatment and Emergency Medicine N. I. Pirogov EAD

    Sofia, 1606

  • University Multiprofile Hospital For Active Treatment Sofiamed OOD

    Sofia, 1797

Other trials in Bulgaria

Germany6 sites

  • Charité Universitaetsmedizin

    Berlin, 15353

  • Universitaetsklinikum Freiburg | Herz-Zentrum Bad Krozingen (Heart Centre Bad Krozingen)

    Freiburg im Breisgau, 79106

  • Medizinische Hochschule Hannover (MHH)

    Hanover, 30625

  • Heidelberg University Hospital

    Heidelberg, 69120

  • Universitätsklinikum Schleswig-Holstein

    Kiel, D-24105

  • Technical University of Munich (TUM)

    München, Bavaria, 81675

Other trials in Germany

Hungary6 sites

  • Semmelweis Egyetem, Varosmajori Sziv- és Ergyogyaszati Klinika

    Budapest, 1122

  • Észak-Pesti Centrumkórház - Honvédkórház

    Budapest, 1134

  • Budapesti Uzsoki Utcai Korhaz

    Budapest, H-1145

  • Somogy Varmegyei Kaposi Mor Oktato Korhaz - Kardiologiai Osztaly

    Kaposvár, 7400

  • Pecsi Tudomanyegyetem Klinikai Kozpont, Szivgyogyaszati Klinika

    Pécs, 7624

  • Szegedi Tudományegyetem, Szent-Györgyi Albert Klinikai Központ - Belgyógyászati Klinika

    Szeged, 6725

Other trials in Hungary

Romania6 sites

  • Spitalul Clinic Judetean De Urgenta Brasov- Sectia Cardiologie 3

    Brasov

  • Spitalul Universitar de Urgenta Militar Central "Dr. Carol Davila", Sectia Clinica Cardiologie I

    Bucharest

  • Spitalul Clinic Judetean De Urgenta Craiova- Sectia de Cardiologie

    Craiova

  • Spitalul Clinic Judetean De Urgenta Targu Mures

    Târgu Mureş, County Mures

  • Spitalul Universitar de Urgenta Bucuresti- Sectia Cardiologie 2

    Târgu Mureş

  • Institutul De Boli Cardiovasculare Timisoara- Clinica de Cardiologie

    Timișoara

Other trials in Romania

Spain6 sites

  • Hospital Universitario de Bellvitge

    Barcelona, Catalonia, 8908

  • Hospital Ramon y Cajal | Cardiology - Research Unit

    Madrid, 28034

  • Universidad Autonoma de Madrid (UAM) - Hospital Universitario La Paz (HULP)

    Madrid, 28046

  • Universidad de Navarra - Clinica Universidad de Navarra (CUN)

    Pamplona, Navarre, 31008

  • Complejo Hospitalario Universitario Santiago de Compostela (CHUS)

    Santiago de Compostela, 15706

  • Hospital Clinico Universitario de Valencia (CHUV)

    Valencia, 46010

Other trials in Spain

United Kingdom5 sites

  • University Hospital of Wales

    Cardiff, CF14 4XW

  • NHS Golden Jubilee

    Glasgow, G81 4DY

  • Leeds General Infirmary

    Leeds, West Yorkshire, LS1 3EX

  • Imperial College London - National Heart & Lung Institute (NHLI) - Royal Brompton Campus

    London, SW3 6LY

  • Manchester Royal Infirmary

    Manchester, Manchester, M13 9WL

Other trials in United Kingdom

Austria4 sites

  • Medizinische Universität Graz

    Graz, 8010

  • Kardiologie & Intensivmedizin Campus III

    Linz, 4021

  • SALK University Hospital

    Salzburg, 5020

  • NÖ Landesgesundheitsagentur

    Sankt Pölten

Other trials in Austria

Netherlands4 sites

  • Amsterdam UMC - Locatie AMC (Academisch Medisch Centrum)

    Amsterdam, North Holland, 1105 AZ

  • Radboud University Medical Center

    Nijmegen, 9101

  • Erasmus Medisch Centrum

    Rotterdam, 3015CE

  • Universitair Medisch Centrum (UMC) Utrecht

    Utrecht, 3508GA

Other trials in Netherlands

Poland3 sites

  • American Heart of Poland S.A. - MCSN, PAKS w Chrzanowie

    Chrzanów, 32-500

  • 1 Wojskowy Szpital Kliniczny z Poliklinika SPZOZ w Lublinie

    Lublin, 20-049

  • Uniwersytecki Szpital Kliniczny (USK) Nr 4 w Lublinie

    Lublin, 20-090

Other trials in Poland

Belgium1 site

  • UZ Antwerpen

    Edegem, 2650

Other trials in Belgium

About this study

Official title: A Phase 2, Adaptive, Double-blinded, Placebo Controlled, Randomized, Multi-center Trial to Evaluate the Efficacy, Safety and Tolerability of Intracoronary Infusion of AB-1002 in Adult Subjects With New York Heart Association (NYHA) Class III Heart Failure and Non-ischemic Cardiomyopathy

This is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial study to evaluate the safety and efficacy of a single dose of AB-1002, administered via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and NYHA Class III symptoms of HF.

Subjects will be randomized into one of three treatment groups in a 1:1:1

Read the detailed description

Subjects will be randomized into one of three treatment groups in a 1:1:1 fashion to either 7.15E13vg (n=30-50), 1.43E14 (n=30-50) or placebo group (n=30-50).

Approximately 90 to 150 subjects will be randomly assigned to study intervention Placebo

Study duration until the primary analysis at 52 week will be approximately 37 months including 25 months of recruitment and 52-week Observation Period after dosing. Once all the subjects complete the 52 weeks Observation Period, the treatment groups will be unblinded and primary analysis performed. Study participation duration: The study will last 52 weeks from dosing, with another 4 years of long-term follow-up for a total of 5 years. During the 4 year long-term follow up sites will contact subjects twice a year for two years, then once a year for the remaining two years for safety, efficacy assessments, and survival

Being tested
AB-1002
Medical subject headings
Heart Failure

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AB-1002Drug

    Also called Gene Therapy, NAN-101

    Intracoronary Infusion of AB-1002 or placebo

Study groups in NCT05598333
Study groupRoleWhat they receive
Treatment Group 1 AB-1002ExperimentalRandomized in 1:1:1 into one of three groups. Group 1: 7.15E13vg/subject (n=30-50)
Receives: Drug: AB-1002
Treatment Group 2 AB-1002ExperimentalRandomized in 1:1:1 into one of three groups. Group 2: 1.43E14vg/subject (n=30-50)
Receives: Drug: AB-1002
Treatment Group 3Placebo comparatorRandomized in 1:1:1 into one of three groups. Group 3: Placebo (n=30-50)
Receives: Drug: AB-1002

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Cardiovascular Related Death

    Measured: 52 weeks

  2. NYHA Classification

    NYHA Classification Change from baseline

    Measured: 52 weeks

  3. Left Ventricular Ejection Fraction

    LVEF change from baseline

    Measured: 52 weeks

  4. Six Minute Walk Test

    6MWT distance change from baseline

    Measured: 52 weeks

Secondary outcomes (1)

  1. Heart Failure Related Hospitalizations

    Number of hospitalizations attributed to heart failure

    Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Oct 2022
  2. Study start20 Oct 2023
  3. Primary completion31 Oct 2026 (estimated)
  4. Study completion31 Dec 2030 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Adaptive, double blinded, placebo controlled, randomized
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
150 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05598333
Sponsor's protocol ID
ASK-CHF2-CS201
Acronym
GenePHIT
Status last verified
Nov 2025
First submitted
25 Oct 2022
Last updated
3 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: No data will be shared with researchers outside of AskBio

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AB-1002 in people with Congestive Heart Failure, run by AskBio Inc at 96 sites in 12 countries.

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 21 inclusion and 25 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: under way, not looking for participants, as of the sponsor's last update on 3 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 96 sites in United States, Canada, Bulgaria, Germany, Hungary and 7 more countries. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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?

Cardiovascular Related Death (time frame: 52 weeks). The study lists 4 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 NCT05598333.

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 3 Dec 2025.

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